The gene therapy is made from the recipient's own blood stem cells, which are modified, and are given back as a one-time, single-dose infusion as part of a hematopoietic (blood) stem cell transplant. [ 3 ]  Prior to treatment, the recipient's own stem cells are collected, and then the recipient must undergo myeloablative conditioning (high-dose chemotherapy), a process that removes cells from the bone marrow so they can be replaced with the modified cells in lovotibeglogene autotemcel. [ 3 ]


The US FDA label contains a black box warning about hematologic malignancy (blood cancer). [ 1 ] [ 3 ]


The safety and effectiveness of lovotibeglogene autotemcel is based on the analysis of data from a single-arm, 24-month multicenter study in participants with sickle cell disease and history of Vaso-occlusive episodes (VOEs) between the ages of twelve and fifty years. [ 3 ]  Effectiveness was evaluated based on complete resolution of VOEs (VOE-CR) between six and eighteen months after infusion with lovotibeglogene autotemcel. [ 3 ]  Twenty-eight (88%) of 32 participants achieved VOE-CR during this time period. [ 3 ]


The FDA granted the application for lovotibeglogene autotemcel  priority review ,  orphan drug ,  fast track , and regenerative medicine advanced therapy designations. [ 3 ]  The FDA granted approval of Lyfgenia to Bluebird Bio Inc. [ 3 ]


The cost effectiveness threshold of the therapy is estimated to be between  US$1.35 million  to $2.05 million. [ 7 ]


Lovotibeglogene autotemcel is the  international nonproprietary name . [ 8 ]


Lovotibeglogene autotemcel is sold under the brand name Lyfgenia. [ 1 ]


Lu 29-252  is a  selective   sigma   σ 2  receptor   ligand  which was under development for the treatment of  anxiety disorders  but was never marketed. [ 1 ] [ 2 ] [ 3 ] [ 4 ] [ 5 ]  It reached the  preclinical stage of development  prior to the discontinuation of its development. [ 1 ]  The drug was under development by  Lundbeck . [ 1 ]


Lu AA41063  is a  selective   adenosine   A 2A  receptor   antagonist . [ 1 ] [ 2 ] [ 3 ] [ 4 ] [ 5 ]   Structurally , it is a non- xanthine . [ 2 ]


The  affinities  (K i ) of the drug for the human  adenosine receptors  are 5.9   nM for the adenosine A 2A  receptor, 410   nM for the adenosine  A 1  receptor  (69-fold lower than for the A 2A  receptor), 260   nM for the adenosine  A 2B  receptor  (44-fold lower than for the A 2A  receptor), and >10,000   nM for the adenosine  A 3  receptor  (>1,695-fold lower than for the A 2A  receptor). [ 2 ]


Lu AA41063 was first described in the  scientific literature  by 2014. [ 1 ] [ 5 ]


Lu AA47070 , a  water-soluble   phosphate   ester   prodrug  of Lu AA41063, is  orally active  and was under development for the treatment of  Parkinson's disease  but was discontinued. [ 6 ] [ 1 ] [ 2 ] [ 7 ] [ 8 ]  In addition to its  antiparkinsonian -like effects, Lu AA47070 reverses  motivational deficits  in animals and hence shows  pro-motivational  effects. [ 9 ] [ 10 ] [ 11 ]


Lu AA47070  is a  selective   adenosine   A 2A  receptor   antagonist  that was under development for the treatment of  Parkinson's disease  but was never marketed. [ 1 ] [ 2 ] [ 3 ]  It has been found to reverse some of the effects of  dopamine   D 2  receptor   antagonists  like  pimozide  and  haloperidol , for instance tremulous jaw movements,  catalepsy ,  locomotor suppression , and other  anti-motivational  effects, in animals. [ 2 ] [ 4 ] [ 5 ]  The drug is a  prodrug  of  Lu AA41063 . [ 6 ] [ 7 ] [ 3 ]  It was discontinued in  phase 1   clinical trials  because it lacked the intended  pharmacological  properties in humans. [ 7 ] [ 1 ]  Lu AA47070 was first described by 2008. [ 8 ]


Lunresertib  is an  investigational new drug  that is being evaluated for the treatment of cancer.  It is an oral small molecule inhibitor of  PKMYT1 , developed by Repare Therapeutics. [ 1 ]  This drug targets cell cycle regulation in tumors with specific genetic alterations, including  CCNE1  amplifications or  FBXW7  and  PPP2R1A  loss of function mutations. It is currently in phase 1/2 clinical trials, both as monotherapy or in combination with  camonsertib , an  ATR  inhibitor. [ 2 ]


Luxdegalutamide , also known as  ARV-766 , is an investigational oral androgen receptor (AR) degrader being developed by Arvinas for the treatment of  metastatic castration-resistant prostate cancer  (mCRPC). [ 1 ] [ 2 ]  It belongs to a class of drugs called  proteolysis targeting chimeras  (PROTACs), which are designed to selectively degrade specific proteins by hijacking the  ubiquitin-proteasome system . [ 3 ]  Luxdegalutamide is a second-generation PROTAC AR degrader that has demonstrated a broader efficacy profile and better tolerability compared to its predecessor, ARV-110, in clinical settings. [ 3 ]  It has shown promise in overcoming resistance associated with certain AR mutations, including the L702H mutation, which is prevalent in up to 24% of treated mCRPC patients. [ 3 ]  As of 2024, luxdegalutamide is being evaluated in phase I/II clinical trials for prostate cancer. [ 1 ] [ 2 ]


LY-266,097  is a research ligand which acts as a  potent  and  selective   antagonist  for the  5-HT 2B   receptor , with more than 100x selectivity over the related  5-HT 2A  and  5-HT 2C  receptor subtypes. [ 1 ]  However, other sources have stated that LY-266,097 is actually a  biased agonist  of the receptor, selective for  G q   signaling  but not  β-arrestin2 . [ 2 ]  The drug has been used to study the role of the 5-HT 2B  receptor in modulating  dopamine  release in the brain, [ 3 ] [ 4 ]  as well as its involvement in other processes such as  allodynia . [ 5 ]


LY-88329  is an  opioid  receptor ligand related to medicines such as  pethidine . It has high affinity to the  μ-opioid receptor , but unlike structurally related drugs such as  3-methylfentanyl  and  OPPPP , LY-88329 is a potent  opioid antagonist . In animal studies it blocks the effects of  morphine  and has  anorectic  action. [ 1 ] [ 2 ]


LY-2109761  is a synthetic compound which acts as a potent and selective inhibitor for the  growth factor   receptor   TGF beta receptor 1 . It is used for research into conditions such as  pulmonary fibrosis  and  cancer . [ 1 ] [ 2 ]


LY-2365109  is a  glycine reuptake inhibitor . It is able to inhibit the  type 1 glycine transporter . [ 1 ]  This inhibition increases extracellular levels of glycine. LY-2365109 has been shown to increase the seizure threshold in mice, meaning that this drug has potential as an anticonvulsant. [ 2 ]


M62812  is a drug which acts as a potent and selective antagonist of  toll-like receptor 4  (TLR4). In animal studies it blocks TLR4-mediated  cytokine  release and has  antiinflammatory  effects, showing efficacy in animal models of  arthritis  and  septic shock . [ 1 ] [ 2 ]


Macitentan/tadalafil , sold under the brand name  Opsynvi , is a  fixed dose combination  medication used for the treatment of  pulmonary arterial hypertension . [ 2 ]  It contains  macitentan , an  endothelin receptor antagonist  (ERA); and  tadalafil , a  phosphodiesterase 5 (PDE5) inhibitor . [ 2 ]


Macitentan/tadalafil was approved for medical use in Canada in October 2021, [ 3 ]  and in the United States in March 2024. [ 4 ] [ 5 ]


Macitentan/tadalafil is  indicated  for the chronic treatment of pulmonary arterial hypertension (PAH, WHO Group I) in adults of WHO functional class (FC) II-III. [ 2 ]


Macitentan/tadalafil may cause harm to the fetus. [ 2 ]


In July 2024, the  Committee for Medicinal Products for Human Use  (CHMP) of the  European Medicines Agency  adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Yuvanci, intended for the treatment of pulmonary arterial hypertension (PAH). [ 6 ]  The applicant for this medicinal product is Janssen-Cilag International NV. [ 6 ]


Magnetic marker monitoring  is a method to monitor the passage of an orally applied  drug  ( tablet ,  capsule , etc.) through the  intestinal tract . The dosage form is enriched with a small amount (0.1 – 2 mg) of  magnetite  (Fe 3 O 4 ), which then is magnetized by a high-energy magnetic field. After application the path of the dosage form can be monitored with special detectors, which contain  Superconducting Quantum Interference Devices  (SQUIDs). Due to the very low magnetic field of the iron oxide a specially shielded room is necessary in order to eliminate environmental magnetic interference. The method should be able to yield information about why tablets dissolve unequally before or after meals, which may be important for the  bioavailability  of drugs.


A more developed method, Advanced Magnetic Marker Monitoring, uses the magnetic capsule, a segmented capsule held together by magnetic forces. The magnetic capsule delivers drugs to any targeted location in the  gastrointestinal tract .


The way the capsule takes is monitored on a PC screen. As soon as the capsule has reached its destination, the magnetic field is destroyed by pressing a button and the drug is released for resorption within milliseconds.


The method is used during development or optimization of drug products and contributes significantly to the reduction in development time and costs.


Areas of use include the determination of resorption windows, early  proof of concept  studies,  interaction  studies, and optimization of pharmaceutical formulations.


Harriet Florence Maling  (née Mylander, October 2, 1918 – March 1987) was an American pharmacologist. [ 1 ] : 833–834   She graduated  Phi Beta Kappa  from  Goucher College  in 1940. [ 2 ]  She went to Harvard Medical School to continue her studies, earning a PhD in 1944.


Her focus of study involved the physiology of the heart, the effects of various drugs upon the heart and on the autonomic and sympathetic nervous systems, experimenting with artificially created myocardial infarction. [ 1 ] : 833–834


Her daughter  Joan Maling  is a linguist and past president of the  Linguistic Society of America .


Mangafodipir  (sold under the brand name  Teslascan  as mangafodipir trisodium) is a  contrast agent  delivered  intravenously  to enhance contrast in  magnetic resonance imaging  (MRI) of the liver, and has potential to serve as an  adjunct  for various  chemotherapeutic  agents and during coronary intervention. It has two parts, a  paramagnetic  manganese(II) ion and the fodipir (dipyridoxyl diphosphate, DPDP)  chelating  agent. When freed from the organic  ligand , the manganese shortens the  longitudinal relaxation  time (T1) in an MRI scan. Normal liver tissue absorbs the manganese more than abnormal or cancerous tissue, which makes the normal tissue appear brighter in MRIs. This enhanced contrast allows lesions to be more easily identified.


Mangafodipir was withdrawn from the US market in 2003 [ 1 ]  and the European market in 2012 for commercial reasons by the manufacturer. [ 2 ]


Reactive oxygen species  (ROS) and  reactive nitrogen species  (RNS) participate in pathological tissue damage.  Mitochondrial  manganese  superoxide dismutase  (MnSOD) normally keeps ROS and RNS in check. During development of mangafodipir as an MRI contrast agent, it was discovered that it possessed MnSOD mimetic activity. Mangafodipir has been tested as a chemotherapy adjunct in cancer patients and as an adjunct to percutaneous coronary intervention in patients with myocardial infarctions, with promising results. [ 3 ]


Mangafodipir has shown promising results in human brain imaging without detectable toxicity [ 4 ] [ 5 ]  and usefulness in detecting lesions in multiple sclerosis. [ 6 ]


Whereas MRI contrast depends on release of Mn 2+ , the MnSOD mimetic activity depends on Mn 2+  that remains bound to DPDP.   Calmangafodipir  [Ca 4 Mn(DPDP) 5 ] (brand name  PledOx )  is stabilized with respect to Mn 2+  and has improved therapeutic activity. [ 3 ]  Calmangafodipir is being explored as a chemotherapy adjunct in cancer patients.


Maridebart cafraglutide  (also known as  MariTide ; [ 1 ]  developmental name  AMG 133 ) is an investigational drug developed by  Amgen  for the treatment of  obesity . It is an agonist of the  GLP-1 receptor  (GLP-1R) and an antagonist of the  glucose-dependent insulinotropic polypeptide receptor  (GIPR). Namely, MariTide consists of a monoclonal antibody against GIPR conjugated to two peptidic GLP-1R agonist molecules via amino acid linkers. [ 2 ]  In a preliminary trial, AMG 133 resulted in a 14.5 percent weight loss after 12 weeks at the highest dose tested. [ 3 ] [ 4 ] [ 5 ]


Masupirdine  is an  investigational new drug  that is being evaluated for the treatment of agitation in  Alzheimer's dementia . [ 1 ]   It is a selective  5-HT 6  receptor  antagonist. [ 2 ]


Mazdutide  (also known as IBI362 or LY3305677) is a dual  agonist  of the  GLP-1 receptor  and  glucagon receptor . It is an analog of  oxyntomodulin  (OXM). [ 1 ] [ 2 ] [ 3 ]  The drug is developed by  Eli Lilly  and is currently in multiple Phase III studies. [ 4 ] [ 5 ]


MB-3  is a drug which acts as a potent and selective inhibitor of the  histone acetyltransferase   enzyme   GCN5 , which usually functions as a negative modulator of  PGC-1α , and so MB-3 acts to indirectly activate PGC-1α. It is used in research into the role of the GCN5/PGC-1α pathway in the regulation of metabolism and cell differentiation. [ 1 ] [ 2 ] [ 3 ]


3',4'-Methylenedioxy-4-methylaminorex  ( MDMAR ) is a  recreational   designer drug  from the  substituted aminorex  family, with  monoamine-releasing effects . [ 1 ] [ 2 ] [ 3 ]  It is a  potent   serotonin–norepinephrine–dopamine releasing agent  (SNDRA). [ 1 ]


MEDI6570  is an antibody of the  lectin-like oxidized low-density lipoprotein receptor-1  (LOX-1) that is being tested in people with type 2 diabetes to see if it reduces their risk of  cardiovascular disease . The drug is developed by  AstraZeneca . [ 1 ] [ 2 ] [ 3 ]


MEDI7352  is an experimental non-opioid  analgesic  drug that works as a bispecific  monoclonal antibody  against  tumour necrosis factor  (TNF) and  nerve growth factor  (NGF); it is developed by  AstraZeneca  for  chronic pain . [ 1 ] [ 2 ] [ 3 ] [ 4 ]


In  toxicology , the  median toxic dose  ( TD 50 ) of a drug or toxin is the dose at which  toxicity  occurs in 50% of cases. [ 1 ] : 73   The  type  of toxicity should be specified for this value to have meaning for practical purposes. The  median toxic dose  encompasses the category of toxicity that is greater than  half maximum effective concentration  ( ED 50 ) but less than the median lethal dose (LD 50 ). However, for some highly potent toxins (ex.  lofentanil ,  botulinum toxin ) the difference between the ED 50  and TD 50  is so minute that the values assigned to them may be approximated to equal doses. Since toxicity need not be lethal, the TD 50  is generally lower than the  median lethal dose  (LD 50 ), and the latter can be considered an  upper bound  for the former. However, since the toxicity is above the effective limit, the TD 50  is generally greater than the ED 50 . If the result of a study is a toxic effect that does not result in death, it is classified as this form of toxicity. [ 2 ]  Toxic effects can be defined differently, sometimes considering the therapeutic effect of a substance to be toxic (such as with  chemotherapeutics ) which can lead to confusion and contention regarding a substance's TD 50 . Examples of these toxic endpoints include cancer, blindness, anemia, and birth defects. [ 2 ]


Medicines reconciliation  or  medication reconciliation  is the process of ensuring that a hospital patient's  medication  list is as up-to-date as possible. It is usually undertaken by a  pharmacist  and may include consulting several sources such as the patient, their relatives or caregivers, or their  primary care physician .


In the  United Kingdom , guidelines on medicines reconciliation are provided by the  National Institute for Health and Care Excellence  (NICE) in collaboration with the  National Patient Safety Agency . [ 1 ]  In accordance with these, it should be carried out within 24 hours of admission to hospital.  From April 2020 it is to be an essential service in the community pharmacy contract in England. [ 2 ]


In the  United States , the  Joint Commission  prioritizes medication reconciliation at hospital admission and during  ambulatory care  as one of the  National Patient Safety Goals . [ 3 ] [ 4 ]


Research has shown that, on average, there is around a 20% discrepancy between medications prescribed on admission to hospital and the true medication list for a given patient. [ 5 ]  Chronic medications are stopped in about 11% of the patients after elective  surgeries [ 6 ]  and 33% of the patients after admission to  intensive care unit . [ 7 ]  The most common omissions are  inhalers  and  analgesia . There are also a small minority of errors in prescribing drugs such as  insulin  or  warfarin , which could have catastrophic consequences including death of the patient.  Pharmacist  involvement help reasons for drug discontinuation being documented [ 8 ]  and  adverse drug reactions  being reconciled in the prescription charts. [ 9 ]  The value of medicines reconciliation is in noticing and correcting these errors before they have a chance to adversely affect the patient concerned. Research shows that the main activity of medicines reconciliation by pharmacists is to identify or assess drug-related problems and discuss them with other professionals. However, the process and the tools used in medicines reconciliation vary greatly. There is a wide variation in how medicines reconciliation is conducted and which methods are utilized in different country and hospitals. [ 10 ]


Meglumine  is a  sugar alcohol  derived from  glucose  that contains an   amino group  modification.  It is often used as an  excipient  in pharmaceuticals [ 1 ]  and in conjunction with iodinated compounds in  contrast media  such as  diatrizoate meglumine ,   iothalamate meglumine , and  iodipamide meglumine . [ 2 ]


Memantine/donepezil , sold under the brand name  Namzaric  among others, is a  fixed dose combination  medication used for the treatment of  dementia  of the  Alzheimer's type . [ 1 ]  It contains  memantine , as the hydrochloride, a  NMDA receptor antagonist ; and  donepezil  as the hydrochloride, an  acetylcholinesterase inhibitor . [ 1 ]  It is taken  by mouth . [ 1 ]


Memantine/donepezil was approved for medical use in the United States in 2014. [ 3 ]


Merotocin  ( INN ) (developmental code name  FE-202767 ), also known as  carba-1-(4-FBzlGly 7 )dOT , is a  peptidic   agonist  of the  oxytocin receptor  that was derived from  oxytocin . [ 1 ] [ 2 ] [ 3 ]  It is under development by  Ferring Pharmaceuticals  for the treatment of  preterm  mothers with lactation failure requiring  lactation  support, and is in  phase II   clinical trials  for this indication. [ 3 ]  Merotocin is potent ( EC 50  < 0.1 nM) and highly selective (>1000-fold over the related  vasopressin receptors ).


A  metallopharmaceutical  is a  drug  that contains a  metal  as an active ingredient. [ 1 ] [ 2 ]  Most commonly metallopharmaceuticals are used as  anticancer  or  antimicrobial  agents. The efficiency of metallopharmaceuticals is crucially dependent on the respective  trace metal  binding forms. [ 3 ]


Examples of metallopharmaceuticals include:


Metaphit  ( 1-[1-(3-Isothiocyanato)phenyl]cyclohexylpiperidine ) is a research chemical that acts as an acylator of  NMDARAn ,  sigma  and  DAT  binding sites in the  CNS . It is the  m -isothiocyanate derivative of  phencyclidine  (PCP) and binds irreversibly (forming a  covalent bond ) to the PCP binding site on the NMDA receptor complex. [ 1 ]  However, later studies suggest the functionality of metaphit is mediated by sites not involved in PCP-induced passive avoidance deficit, and not related to the NMDA receptor complex. [ 2 ]  Metaphit was also shown to prevent d-amphetamine induced hyperactivity, while significantly depleting dopamine content in the nucleus accumbens. [ 3 ]  Metaphit was the first acylating ligand used to study the  cocaine  receptor. [ 4 ]  It is a structural isomer of the similar research compound  fourphit , as it and metaphit both are isothiocyanate substituted derivatives of an analogous scaffold shared with PCP. [ 5 ]


Methimepip  is a  histamine agonist  which is highly selective for the  H 3  subtype. It is the  N - methyl  derivative of  immepip . [ 1 ]


Methiodal  is a pharmaceutical drug that was used as an  iodinated contrast  medium for  X-ray imaging . [ 1 ]  Its uses included  myelography  (imaging of the  spinal cord ); for this use, cases of adhesive  arachnoiditis  have been reported, similar to those seen under the contrast medium  iofendylate . [ 2 ]


It is not known to be marketed anywhere in the world in 2021. [ 3 ]


Methoxyetomidate  is an investigational anesthetic agent being developed by Jinzhou Ahon Pharmaceutical Co., Ltd. It is a short-acting intravenous  anesthetic  that acts as a  positive allosteric modulator  of  GABAA receptors . [ 1 ] [ 2 ]  As of 2024, methoxyetomidate is undergoing Phase 3 clinical trials for use in anesthesia. [ 3 ] [ 1 ]


3,4-Methylenedioxyphenmetrazine , also known as  3-MDPM , is a recreational  designer drug  with  stimulant  effects. It is a  substituted phenylmorpholine  derivative, closely related to better known drugs such as  phenmetrazine  and  3-fluorophenmetrazine . It has been identified as a synthetic impurity formed in certain routes of  MDMA  manufacture. [ 1 ]


3,4-Methylenedioxyphenylpropan-2-one [ 1 ]  or  piperonyl methyl ketone  ( MDP2P  or  PMK ) is a  chemical compound  consisting of a  phenylacetone   moiety   substituted  with a  methylenedioxy   functional group . It is commonly  synthesized  from either  safrole  (which, for comparison, is 3-[3,4-(methylenedioxy)phenyl]-2-propene) or its  isomer   isosafrole  via  oxidation  using the  Wacker oxidation  or  peroxyacid  oxidation methods. MDP2P is unstable at room temperature and must be kept in the freezer in order to be preserved properly.


MDP2P is a  precursor  in the  chemical synthesis  of the  methylenedioxyphenethylamine  (MDxx)  class  of compounds, the classic example of which is  3,4-methylenedioxy- N -methylamphetamine  (MDMA), and is also an  intermediate  between the MDxx family and their slightly more distant precursor safrole or isosafrole. On account of its relation to the MDxx chemical class, MDP2P, as well as safrole and isosafrole, are in the  United States  (U.S.)  Drug Enforcement Administration  (DEA)  List I of Chemicals  of the  Controlled Substances Act  (CSA) via the  Chemical Diversion and Trafficking Act  (CDTA). It is also considered a category 1 precursor in the European Union.


4-Methylphenmetrazine  ( mephenmetrazine ,  4-MPM ,  PAL-747 ) is a recreational  designer drug  with  stimulant  effects. It is a  substituted phenylmorpholine  derivative, closely related to better known drugs such as  phenmetrazine  and  3-fluorophenmetrazine . It was first identified in Slovenia in 2015, [ 1 ] [ 2 ]  and has been shown to act as a monoamine releaser with some preference for  serotonin  release. [ 3 ] [ 4 ]


Metrizoic acid  is a  pharmaceutical drug  that was used as an  iodinated contrast  medium for  X-ray imaging . Its uses included  angiography [ 1 ]  (imaging of blood vessels and  heart chambers ) and  urography [ 2 ]  (imaging of the  urinary tract ), but it has been discontinued, at least in the US. [ 3 ]


It was used in form of its salts,  metrizoates . Due to its high  osmolality , metrizoic acid had a risk of inducing allergic reactions higher than that of lower osmolar contrast media. [ 4 ]


The iodine content of metrizoate ranged from 370 mg/ml to 440 mg/ml, with osmolarity has high as 2100 mOsm/kg. The viscosity is 3.4 cP at 37 degree Celsius (human body temperature). [ 5 ]


Side effects of metrizoate are: urticaria, headache, nausea, vomiting, dizziness, and hypotension. Other side effects include minor electrocardiographic changes such as tachycardia, bradycardia, and inversion of T waves. [ 6 ]


Microtrol  is an extended-release drug delivery system. [ 1 ]  It is used in certain medications like  Adderall  and incorporates two types of beads. The first dissolves immediately, releasing half of the medication, while the second type dissolves much more slowly releasing the remaining medication four hours later. Maximum plasma concentration is achieved in seven hours, compared to instant-release which reaches maximum concentration in three hours. [ citation needed ]  Microtrol is manufactured by Supernus Pharmaceuticals, Inc. [ 2 ]


Milademetan  is an  investigational new drug  that is being evaluated to treat  liposarcoma . [ 1 ]  It is a  MDM2  inhibitor. [ 2 ]


Miotine  is an  anticholinesterase  drug. Miotine was the first synthetic  carbamate  that was used clinically. [ 2 ]


Unlike the miotine analog  neostigmine , it doesn't have a quaternary  ammonium group  to give it a permanent positive charge. It can exist as an uncharged  free base  which could allow it to cross the  blood–brain barrier  and cause unwanted  central nervous system  (CNS) side effects. [ 3 ]


Mitiperstat  (AZD4831) is an irreversible inhibitor of  myeloperoxidase  and experimental drug in development for  heart failure with preserved ejection fraction . [ 1 ] [ 2 ] [ 3 ] [ 4 ] [ 5 ] [ 6 ] [ 7 ]  It is being developed by  AstraZeneca . [ 8 ]


Mitoquinone mesylate  ( MitoQ ) is a synthetic analogue of  coenzyme Q10  which has  antioxidant  effects. It was first developed in New Zealand in the late 1990s. [ 1 ]  It has significantly improved bioavailability and improved  mitochondrial  penetration compared to coenzyme Q10, [ 2 ] [ 3 ]  and has shown potential in a number of medical indications, [ 4 ] [ 5 ] [ 6 ] [ 7 ]  being widely sold as a  dietary supplement . [ 8 ] [ 9 ]


A 2014 review found insufficient evidence for the use of mitoquinone mesylate in  Parkinson's disease  and other movement disorders. [ 10 ]


Mitotropes  are a novel class of drugs that aim to improve cardiac performance by influencing the  mitochondria . Their intended effect is similar to the calcium-based  inotropes , and intend to have fewer long-term side effects. [ 1 ]


MK-3984  is a  drug  which acts as a  selective androgen receptor modulator  (SARM).  It is no longer being developed. [ 1 ]


MK-4541  is a dual  selective androgen receptor modulator  (SARM) and  5α-reductase inhibitor  (5α-RI) which has been of interest for the potential treatment of  prostate cancer  but has not been marketed at this time. [ 2 ] [ 3 ] [ 1 ] [ 4 ] [ 5 ] [ 6 ]  It is intended for use  by mouth . [ 1 ]


The drug is a  steroidal   androgen receptor  (AR)  modulator  with mixed  agonistic  ( androgenic ) and  antagonistic  ( antiandrogenic ) effects. [ 7 ] [ 8 ] [ 4 ] [ 5 ] [ 6 ]  In  preclinical research  and  animal studies , MK-4541 has been found to have androgenic or  anabolic  effects in  muscle  and  bone , to strongly suppress  testosterone  levels (likely via androgenic  antigonadotropic  effects), and to have antiandrogenic effects in the  prostate  and in  prostate cancer   cells . [ 7 ] [ 8 ] [ 4 ] [ 5 ] [ 6 ]   Structurally , it is specifically a  4-azasteroid   derivative . [ 2 ]


MK-4541 was first described in the  scientific literature  by 2014. [ 5 ] [ 6 ]  It was identified by screening of 3,000 compounds that were manually designed and predicted to have SARM activity. [ 9 ] [ 5 ]  The drug was developed by  Merck . [ 3 ]  It might be being developed for potential medical use, but its developmental status has not been publicly disclosed. [ 3 ]  In any case, MK-4541 is not known to have advanced past preclinical studies as of 2020. [ 1 ]


ML2-SA1  ( EVP-22 ) is a chemical compound which acts as an "agonist" (i.e. channel opener) for the  TRPML2  calcium channel, with high selectivity for TRPML2 and no significant activity at the related  TRPML1  and  TRPML3  channels. It has been used to demonstrate the role of TRPM2 in  immune system  function, both triggering release of the  chemokine   CCL2  from  macrophages  and stimulating macrophage migration and endolysosomal trafficking. [ 1 ] [ 2 ]


Mochida Pharmaceutical Co., Ltd  ( 持田製薬株式会社 ,  mochida seiyaku kabushiki gaisha )  is a Japanese  pharmaceutical   company . [ 1 ]


One of its products is the antidepressant  setiptiline  (Tecipul). [ 2 ]


This article about a Japanese corporation- or company-related topic is a  stub . You can help Wikipedia by  expanding it .


In  pharmacology  and  biochemistry ,  mode of action  ( MoA ) describes a functional or anatomical change, resulting from the exposure of a living organism to a substance. [ 1 ]  In comparison, a  mechanism of action  (MOA) describes such changes at the molecular level. [ 2 ] [ 1 ]


A mode of action is important in classifying chemicals, as it represents an intermediate level of complexity in between molecular mechanisms and physiological outcomes, especially when the exact molecular target has not yet been elucidated or is subject to debate. A mechanism of action of a chemical could be "binding to DNA" while its broader mode of action would be "transcriptional regulation". [ 3 ]  However, there is no clear consensus and the term mode of action is also often used, especially in the study of  pesticides , to describe molecular mechanisms such as action on specific  nuclear receptors  or  enzymes . [ 4 ] [ 5 ] [ 6 ] [ 7 ] [ 8 ] [ 9 ] [ 10 ]  Despite this, there are classification attempts, such as the HRAC's  classification  to manage  pesticide resistance . [ 11 ]


Model-Informed Precision Dosing  (MIPD for short) is the use of  pharmacometric  models with  computer software  to optimize drug dosage for an individual patient. [ 1 ]


Developed in the late 1960s under the impetus of clinical pharmacologists such as Lewis Sheiner and Roger Jelliffe, these approaches involve applying the equations and parameters describing a drug's  pharmacokinetics  and  pharmacodynamics  to define the best dosage regimen for a given individual, likely to produce circulating concentrations associated with maximum efficacy and minimum toxicity. Models typically take into account the patient's demographic characteristics (age, gender, ethnicity), clinical profile (body measurements, renal and hepatic function, comorbidities, co-medications, dietary habits, substances use) and possibly genetic factors (e.g. polymorphisms affecting  cytochromes  or  drug transporters ). When starting a treatment, these models can be used to select  a priori  the optimal dosage for a patient, based on simulations. During the treatment course, these same models can be used to integrate the results of  Therapeutic Drug Monitoring  (i.e. the measurement and medical interpretation of circulating drug concentrations) or the measurement of biomarkers of efficacy or toxicity, in an  a posteriori  approach to dose optimization, derived from  Bayesian inference  and  feedback loops . Practically, these approaches make extensive use of computer software dedicated to the clinical use of  pharmacokinetic/pharmacodynamic models , belonging to the  computerized clinical decision support tools . [ 2 ] [ 3 ]  They complement  Model-Informed Drug Development  (MIDD), which is mainly carried out by pharmaceutical industry researchers prior to marketing.


Prescribers are expected to make increasingly regular use of model-driven precision dosing tools for patient treatment and follow-up. Dosage individualization represents the quantitative aspect of  precision medicine , while the qualitative aspect lies in the personalized choice of the best drug to treat a given pathology. This optimization of dose selection is especially desirable for drugs with narrow  therapeutic index  (i.e. effective concentration close to toxic ones). It is also important when a treatment is to be applied to patients with peculiarities, such as children, frail elderly persons, polymorbid patients or those already heavily treated. Technical hurdles still limit the wide implementation of these approaches in clinical practice, but it is to be expected that  electronic patient records  will pursue their development, thus enabling the increasing integration of model-informed precision dosing into medical practice. [ 4 ] [ 5 ]


Monastrol  is a cell-permeable small molecule inhibitor discovered by Thomas U. Mayer in the lab of  Tim Mitchison . Monastrol was shown to inhibit the  kinesin -5 (also known as  KIF11 , Kinesin Eg5), a  motor protein  important for spindle bipolarity. [ 1 ]


Monastrol binds to a long loop that is specific to the Eg5 (also known as  KIF11  or kinesin-5) kinesin family, and allosterically inhibits ATPase activity of the kinesin  [ 2 ]


Moprolol  is a  beta-adrenergic antagonist ,  or  beta blocker . It is typically prescribed to treat hypertension, high blood pressure, angina pectoris, arrhythmias, anxiety, and glaucoma. [ 1 ]


Moprolol is currently off the market, most likely due to the manufacturer being in violation of US good manufacturing practices. [ 1 ]


MRTX1133  is an  investigational drug  that targets the G12D mutation in  KRAS  dependent cancers. [ 1 ] [ 2 ]   It is currently in a phase 1/2 clinical trial for the treatment of solid tumors. [ 3 ]  MRTX1133 is considered to be harmful from direct skin or eye exposure other than transient irritation. It may cause irritation of the respiratory system if inhaled. [ 4 ]


MSX-2  is a  selective   adenosine   A 2A  receptor   antagonist  used in  scientific research . [ 1 ]  It is a  xanthine  and a  derivative  of the  non-selective   adenosine receptor antagonist   caffeine . [ 1 ] [ 2 ]


The  affinities  (K i ) of MSX-2 for the human  adenosine receptors  are 5.38 to 14.5   nM for the adenosine A 2A  receptor, 2,500   nM for the adenosine  A 1  receptor  (172- to 465-fold lower than for the A 2A  receptor), and >10,000   nM for the adenosine  A 2B  and  A 3  receptors  (>690-fold lower than for the A 2A  receptor). [ 3 ] [ 4 ]


MSX-2 has poor  water solubility , which has limited the use of MSX-2 itself. [ 1 ] [ 5 ]  Water-soluble  ester   prodrugs  of MSX-2, including  MSX-3  (a  phosphate  ester prodrug) and  MSX-4  (an  amino acid  ester prodrug), have been developed and used in place of MSX-2. [ 1 ] [ 5 ]  MSX-3 is best-suited for use by  intravenous administration , whereas MSX-4 can be administered by  oral administration . [ 5 ] [ 6 ]


MSX-3 and MSX-4 reverse  motivational deficits  in animals and hence have the capacity to produce  pro-motivational  effects. [ 7 ] [ 8 ] [ 9 ]


MSX-2 and MSX-3 were first described in the  scientific literature  by 1998. [ 10 ] [ 11 ]  Subsequently, MSX-4 was developed and described by 2008. [ 5 ] [ 6 ]


MSX-4  is a  selective   adenosine   A 2A  receptor   antagonist  used in  scientific research . [ 1 ]  It is a  water-soluble   amino acid   ester   prodrug  of  MSX-2 , the  active metabolite  of the drug. [ 2 ] [ 1 ]  MSX-4 reverses the  motivational deficits  induced by the  dopamine   D 2  receptor   antagonist   eticlopride  in animals and hence has the capacity to produce  pro-motivational  effects. [ 3 ] [ 4 ]  MSX-4 was first described in the  scientific literature  by 2008. [ 1 ]


Heinrich Mückter  (14 June 1914 – 22 May 1987) was a German medical doctor, pharmacologist and chemist.


During the Nazi occupation of Poland, Mückter was deputy director of the Kraków Institute for Typhus and Virus Research. Mückter and his colleagues repeatedly experimented on  concentration camp  prisoners in  Buchenwald . Many prisoners died as a result of the experiments.


Accused by Polish war crimes prosecutors of conducting medical experiments on concentration camp prisoners and Nazi forced labourers, Mückter escaped arrest and fled back to Germany. [ 1 ]


In 1946 Mückter became Head of Research at the  Grünenthal  pharmaceutical company, where he further developed the infamous drug  thalidomide  which had been synthesized in 1952 by Chemical Industry Basel. Aggressively-marketed as an over-the-counter sleeping pill and remedy for morning sickness in pregnancy, thalidomide was first made available on 1 October 1957, and it became the second best-selling medication in Germany after Bayer Aspirin. Thalidomide was eventually found to cause  miscarriages, severe birth defects in babies whose mothers had taken the medication while pregnant, and severe nerve damage . [ 1 ] [ failed verification ] [ 2 ]


In January 1968, Mückter was put on trial along with other Grünenthal employees. The trial ended abruptly in April 1970 with a settlement. [ citation needed ]


Mückter was never charged in relation to his role in experiments on concentration camp prisoners, nor his role in the thalidomide scandal. He died on 22 May 1987. [ citation needed ]


This article about a German chemist is a  stub . You can help Wikipedia by  expanding it .


This article related to  Nazi Germany  is a  stub . You can help Wikipedia by  expanding it .


The  Multistate Pharmacy Jurisprudence Examination  ( MPJE ) is a  pharmacy  law examination created by the  National Association of Boards of Pharmacy  (NABP) in the United States to help individual state boards of pharmacy assess the competency and knowledge of  pharmacy  law. [ 1 ]  It is required as a prerequisite for a pharmacy license by 48 states and the  District of Columbia . Arkansas, California, and  Idaho  utilize state administered examinations.


Murodermin  ( INN ), also known as  recombinant murine epidermal growth factor  ( rmEGF ), is a  recombinant  form of  mouse   epidermal growth factor  (EGF) and an  EGF receptor   agonist  which was never marketed. [ 1 ] [ 2 ]


N-0385  is an experimental  small molecule   TMPRSS2 -inhibitor being investigated for its potential use in the prevention and treatment of  COVID-19 . [ 1 ] [ 2 ] [ 3 ] [ 4 ] [ 5 ]


N-0385 is thought to have antiviral effects by targeting key proteins involved in the viral entry process, including TMPRSS2,  ACE2 , and  DPP4 . By interfering with the interactions between these proteins and the  SARS-CoV-2   spike protein , N-0385 effectively blocks the virus from gaining access to host cells. [ 3 ] [ 4 ] [ 5 ]  Additionally, N-0385 appears to modify the immune responses and inflammatory pathways associated with the infection by regulating  TLR7 ,  NLRP3 , and  IL-10 , potentially reducing the severity of COVID-19 symptoms and reducing tissue damage associated with the infection. [ 4 ]


N-Ethyl-2C-B  is a recreational  designer drug  with  psychedelic  effects. [ 1 ]  It was first synthesised in the 1990s, [ 2 ]  and was first identified as a new psychoactive substance in Finland in 2007. [ 3 ]  It is specifically listed as an illegal drug in Finland, [ 4 ]  and controlled under analogue provisions in a number of other jurisdictions.


Nacubactam  is an investigational  β-lactamase inhibitor  being developed for the treatment of  infections  caused by  carbapenem-resistant Enterobacteriaceae  (CRE). It belongs to the  diazabicyclooctane  (DBO) class of compounds and exhibits a dual mechanism of action. Nacubactam inhibits serine β-lactamases, including classes A and C and some class D enzymes, while also directly inhibiting  penicillin-binding protein 2  (PBP2) in  Enterobacteriaceae . [ 1 ] [ 2 ]  This unique profile allows nacubactam to both protect partner β-lactam antibiotics from degradation and exert direct antibacterial effects. As of 2024, nacubactam is undergoing clinical trials in combination with other β-lactam antibiotics such as  meropenem ,  cefepime , and  aztreonam  for the treatment of complicated  urinary tract infections  and other serious bacterial infections caused by multidrug-resistant pathogens. [ 3 ] [ 4 ]


Nadofaragene firadenovec , sold under the brand name  Adstiladrin , is a  gene therapy  for the treatment of  bladder cancer . [ 1 ] [ 3 ] [ 4 ]  It is a non-replicating (cannot multiply in human cells)  adenoviral  vector-based gene therapy. [ 3 ]


The most common adverse events including laboratory abnormalities, include increased glucose, instillation site discharge, increased triglycerides, fatigue, bladder spasm, micturition urgency, increased creatinine, hematuria, decreased phosphate, chills, dysuria, and pyrexia. [ 4 ]


Nadofaragene firadenovec was approved for medical use in the United States in December 2022. [ 3 ] [ 4 ] [ 5 ]


Nadofaragene firadenovec is  indicated  for the treatment of adults with high-risk  Bacillus Calmette-Guérin  (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. [ 1 ] [ 3 ]


The safety and effectiveness of nadofaragene firadenovec was evaluated in a multicenter clinical study (Study CS-003 (NCT02773849)) that included 157 participants with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer, 98 of whom had BCG-unresponsive carcinoma in situ with or without papillary tumors and could be evaluated for response. [ 3 ] [ 4 ]  Participants received nadofaragene firadenovec once every three months for up to twelve months, or until unacceptable toxicity to therapy or recurrent high-grade non-muscle-invasive bladder cancer. [ 3 ] [ 4 ]  Overall, 51% of enrolled participants using nadofaragene firadenovec therapy achieved a complete response (the disappearance of all signs of cancer as seen on cystoscopy, biopsied tissue, and urine). [ 3 ]  The median duration of response was 9.7 months. [ 3 ]  Forty-six percent of responding participants remained in complete response for at least one year. [ 3 ]  The major efficacy outcome measures were complete response at any time and duration of response. [ 4 ]  Complete response was defined as negative cystoscopy with applicable transurethral resection of bladder tumor and biopsies and urine cytology. [ 4 ]  Random bladder biopsies of five sites were conducted in participants remaining in complete response at twelve months. [ 4 ]


The US  Food and Drug Administration  (FDA) granted the application for nadofaragene firadenovec  priority review ,  breakthrough therapy ,  fast track , and  orphan drug  designations. [ 3 ] [ 4 ]  The FDA granted approval of Adstiladrin to  Ferring Pharmaceuticals  A/S. [ 3 ]


Nadofaragene firadenovec is the  international nonproprietary name  (INN). [ 6 ]


National Association of Boards of Pharmacy  ( NABP ) is a  501(c)(3)   nonprofit organization  that assists member boards of pharmacy for the purpose of protecting public health. [ 3 ]  It has 54 active members and 12 associate members. [ 6 ]  Active member boards include all 50 United States, the District of Columbia, Guam, Puerto Rico, and the Virgin Islands. Associate member boards are The Bahamas, and 10 Canadian provinces. [ 6 ]  Australia was formerly an associate member but was removed in 2020. [ 7 ]


NABP supports its member boards of pharmacy by offering: [ 8 ]


Representatives of twenty [ a ]  state and territorial boards of pharmacy met at the  Coates House Hotel  in Kansas City, Missouri, on September 7, 1908. At the meeting, they formed the National Association of Boards of Pharmacy that would provide for interstate reciprocity in pharmaceutical licenses based on a uniform minimum standard of education and uniform legislation. [ 1 ] [ 9 ]  It was intended to act independently from the  American Pharmaceutical Association , as it would help resolve differences between state boards of pharmacy. [ 1 ]  NABP's  constitution  stated that the NABP would become operative when ten state or territorial boards of pharmacy had adopted the rules and regulations that had NABP adopted. [ 1 ]  George Reimann was elected its first  president , and J. M. A. Laue was elected its first  vice president . [ 9 ]


NABP was  incorporated  in Kentucky on November 5, 1913. [ 10 ]  Its original governing board consisted of J. W. Gayle, E. Berger, M. G. Beehe, John Culley, and J. J Campbell. [ 10 ]


In 1999, the NABP developed the Verified Internet Pharmacy Practice Sites (VIPPS) program to accredit online pharmacies. [ 11 ]  NABP says it looks at an online pharmacy's state licenses, its state inspection reports, and its procedures for dispensing of drugs when determining whether to accredit an online pharmacy, and it requires the online pharmacy to adhere to the VIPPS criteria and program requirements. [ 11 ]


In 2004, the NABP developed the  Verified-Accredited Wholesale Distributors  (VAWD) program to accredit Wholesale Distributors and to help protect the public from the threat of counterfeit drugs. [ 12 ]


In 2011, the NABP implemented a data exchange that allows authorized pharmacists, law enforcement agents, and regulatory boards to access patient-specific controlled substance prescription information. Known as  NABP PMP InterConnect® , the platform provides a single page summary of patient's drug seeking activities across state lines. As of 2018, authorized users in 42 states have accessed data using the system in order to prevent drug abuse and drug diversion. [ 13 ]


In 2014, the NABP launched the  generic top-level domain  (gTLD)  .pharmacy , "to provide consumers around the world a means for identifying safe, legal, and ethical online pharmacies and related resources". [ 14 ]


The impartiality of the domain has been questioned, because  Eli Lilly and Company ,  Merck & Co. , and  Pfizer  are the main contributors to the NABP application.  Previously, that application was challenged by  Public Citizen ,  Knowledge Ecology International , and the  Canadian International Pharmacy Association . [ 15 ]


Navafenterol  is an  investigational drug  that had been evaluated for  chronic obstructive pulmonary disease . [ 1 ]  It is a  Beta2 agonist  and a  muscarinic antagonist . Further development has been discontinued for strategic reasons. [ 2 ]


Nebivolol/valsartan , sold under the brand name  Byvalson  among others, is a  medication  used to treat  hypertension . [ 1 ] [ 2 ]


It is available as a  generic medication . [ 3 ]


Trans -NED-19  is a drug which acts as a potent and selective antagonist of the endogenous calcium channel opener  nicotinic acid adenine dinucleotide phosphate  (NAADP), thereby reducing the normal NAADP-mediated calcium flux without blocking calcium channels directly. It is used in research into the functions of NAADP signalling inside many different cell types. [ 1 ] [ 2 ] [ 3 ] [ 4 ] [ 5 ] [ 6 ] [ 7 ] [ 8 ]


Nemtabrutinib  (MK-1026, formerly ARQ 531) is a small molecule drug that works as a reversible  Bruton's tyrosine kinase  (BTK) inhibitor; unlike other BTK inhibitors it also works against some mutated forms of BTK. [ 1 ] [ 2 ] [ 3 ] [ 4 ] [ 5 ]   Merck  paid $2.7 billion to acquire the company ArQule and the drug, which is being investigated as a cancer treatment. [ 6 ]


Neo-Viagra-Y.R.  ( Korean :  네오비아그라-Y.R.(청춘부활))  is a pharmaceutical product produced by the  Korea Oriental Instant Medicinal Centre , a state-owned North Korean company. [ 1 ]  The product was developed in 2005, by north Korean doctor Ryu Il nam. [ 2 ]
Although marketed as a  herbal   health supplement  based in  Koryo medicine  ( traditional Korean medicine  promoted by North Korea), analysis of the product shows that its  active ingredient  is 50 mg of  Sildenafil  (the lowest dose of  Pfizer 's genuine Viagra tablets), though used in a different formulation to genuine Viagra tablets. [ 3 ]   As the drug has not been independently clinically tested, it may cause serious side effects such as a  heart attack  or death. [ 4 ]


The product is marketed as medicine which can cure  sexual dysfunction  in both men and women, cure  hepatitis , [ 5 ]  ease  bone pain , cure  kidney dysfunction  and  arteriosclerosis , with testimonials describing it as "the magic medication of the 21st century". [ 1 ]  Promotional material for the product describes it as "the best  sexual function  activator at the moment" [ 6 ]  which is "officially recognized in many countries for its excellent effect in immediately increasing stamina and it is believed to be better than American Sildenafil (Viagra)". [ 3 ]


It is believed to be primarily targeted for export, as a means for the  North Korean government  (which is  subject to economic sanctions ) to earn foreign currency. It is sold by websites based in China and Russia. [ 3 ]  In 2017, police in  Busan , South Korea, arrested three Russian nationals for illegally selling North Korean Sildenafil and Neo-Viagra-Y.R. tablets in South Korea. [ 7 ]


Neostigmine/glycopyrronium bromide , sold under the brand name  Prevduo  , is a fixed-dose  combination medication  used for the reversal of the effects of  non-depolarizing   neuromuscular blocking agents  after  surgery . [ 2 ]  It contains  neostigmine  as the methylsulfate, a  cholinesterase inhibitor , and  glycopyrronium bromide , an  antimuscarinic agent . [ 2 ]


Neostigmine/glycopyrronium bromide was approved for medical use in the United Kingdom in 2007, [ 1 ]  and in the United States in February 2023. [ 3 ]


Neostigmine/glycopyrronium bromide is  indicated  for the reversal of the effects of non-depolarizing neuromuscular blocking agents after surgery, while decreasing the peripheral muscarinic effects (e.g., bradycardia and excessive secretions) associated with cholinesterase inhibition following non-depolarizing neuromuscular blocking agent reversal administration. [ 3 ]


Nexafed  is a formulation of  pseudoephedrine  developed by  Acura Pharmaceuticals  used to deter the use of the pseudoephedrine contained in the product for illicit  methamphetamine  synthesis. [ 1 ]  Nexafed uses a polymer matrix that forms a thick gel when hydrated, preventing the extraction of pseudoephedrine. [ 2 ]  Nexafed is the only meth-resistant formulation of pseudoephedrine that shows therapeutic equivalence to Sudafed. [ 3 ]


It is sold at various pharmacies in the US, including Fruth Pharmacy [ 4 ] [ 5 ]  and  Kroger , particularly in communities where illicit methamphetamine production is an issue. [ citation needed ]


Nibrozetone  is an  investigational new drug  that is being evaluated by  EpicentRx  for the treatment of oral  mucositis  in  head and neck cancer  patients.  It is a small molecule that combines direct inhibition of the  NLRP3   inflammasome , induction of  NRF2 , and release of  nitric oxide  under  hypoxic  conditions. [ 1 ] [ 2 ]  It has received  Fast Track  designation from the FDA for severe oral mucositis in head and neck cancer patients. [ 3 ]


A  nicotine nasal spray  is a  nasal spray  that contains a small dose of  nicotine , which enters the  blood  by being absorbed through the lining of the nose. This helps stop nicotine  cravings  and relieves symptoms that occur when a person is trying to  quit smoking . A  prescription  is needed for nicotine nasal spray in many countries. In the  United Kingdom , it can be purchased in a pharmacy as an  over-the-counter drug .


This article incorporates  public domain material  from  Dictionary of Cancer Terms .  U.S. National Cancer Institute .


Nidufexor  ( LMB-763 ) is a drug which acts as a  partial agonist  of the  farnesoid X receptor  (FXR). It has reached Phase II  clinical trials  for the treatment of  diabetic nephropathy  and  nonalcoholic steatohepatitis . [ 1 ] [ 2 ]


Nifene  is a high affinity, selective nicotinic α4β2* receptor partial agonist used in medical research for  nicotinic acetylcholine receptors , usually in the form of  nifene ( 18 F ) [ 1 ] [ 2 ]  as a  positron emission tomography  (PET)  radiotracer . [ 3 ] [ 4 ]


Nifene has been used to assess the efficacy of acetylcholinesterase inhibitors in animal models, because the neurotransmitter acetylcholine competes with the binding of nifene at the nicotinic receptor site. [ 5 ] [ 6 ]   Learning and behavior studies in animal models using nifene have suggested a potential role of the nicotinic receptors located in distinct white matter tracts. [ 7 ]   Nifene studies in animal models of lung cancer have suggested an upregulation of the nicotinic receptor in the lung tumors. [ 8 ] [ 9 ]   Novel PET and SPECT imaging agents as potential  receptor antagonists  have been developed based on the structure of nifene; niodene for SPECT, [ 10 ]  nifrolene for PET  [ 11 ]  and niofene for PET/SPECT. [ 12 ]  These new derivatives take advantage of the unique  in vivo  imaging properties of nifene. [ 13 ]  Human studies with ( 18 F )-nifene make it a promising nicotinic α4β2* receptor PET radiotracer for scientific research and has exhibited reliable test-retest reproducibility. [ 14 ]  Human white matter thalamic radiations (or tracts) were well demarcated and quantified using ( 18 F )-nifene. [ 15 ]


( R )-3-Nitrobiphenyline  is a drug which acts as an  α 2 -adrenergic   agonist , selective for the  α 2C  subtype, as well as being a weak  antagonist  at the  α 2A  and  α 2B  subtypes. It has been used in scientific research to characterize the binding and functional properties of the  α 2C  subtype. [ 1 ] [ 2 ]


Nitrosoproline  is a  nitroso   derivative  of the  amino acid   proline .


Nivolumab/hyaluronidase , sold under the brand name  Opdivo Qvantig , is a  fixed-dose combination   anti-cancer medication  used for the treatment of various forms of  cancer . [ 1 ] [ 2 ]  Nivolumab/hyaluronidase contains  nivolumab , a  programmed death receptor-1  (PD-1)-blocking  monoclonal antibody ; and  hyaluronidase , an  endoglycosidase . [ 1 ]  It is given by  subcutaneous injection . [ 1 ] [ 2 ]


Nivolumab/hyaluronidase was approved for medical use in the United States in December 2024. [ 2 ]


In December 2024, the US  Food and Drug Administration  (FDA) approved the combination of nivolumab and  hyaluronidase  across approved adult, solid tumor nivolumab  indications  as monotherapy, monotherapy maintenance following completion of hyaluronidase plus  ipilimumab  combination therapy, or in combination with  chemotherapy  or  cabozantinib . [ 2 ]  The approval includes indications for renal cell carcinoma, melanoma, non-small cell lung cancer, head and neck squamous cell carcinoma, urothelial carcinoma, colorectal cancer, hepatocellular carcinoma, esophageal carcinoma, gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma. [ 2 ]


The subcutaneous injection of nivolumab and hyaluronidase was evaluated in CHECKMATE-67T (NCT04810078), a multicenter, randomized, open-label trial in participants with advanced or metastatic clear cell renal cell carcinoma who received no more than two prior systemic treatment regimens. [ 2 ]  A total of 495 participants were randomized to receive either subcutaneous nivolumab and hyaluronidase or intravenous nivolumab. [ 2 ]


Nivolumab/hyaluronidase was approved for medical use in the United States in December 2024. [ 2 ] [ 3 ]


NJK14047  is a drug which acts as a selective inhibitor of the enzyme  p38 mitogen-activated protein kinase  (p38 MAPK). It has  antiviral  and  antiinflammatory  effects and was originally developed as a potential treatment for inflammatory lung conditions such as  influenza . Subsequent research has also shown promise for various other conditions in which inflammation plays a role, including  Alzheimer's disease ,  asthma ,  arthritis  and  psoriasis . [ 1 ] [ 2 ] [ 3 ] [ 4 ]


NMDA receptor modulators  ( glutamate  modulators) are a new form of  antipsychotic  that are in Phase II FDA studies. The first compound studied was  glycine  which was hypothesized by Daniel Javitt after observation that people with  phencyclidine (PCP)-induced  psychosis  were lacking in  glutamate  transmission. [ 1 ]  (PCP is an  NMDA receptor antagonist  that blocks glutamate.) In giving glycine to people with PCP-induced psychosis a recovery rate was noted. From there, it was hypothesized that people with psychosis from  schizophrenia  would benefit from increased glutamate transmission and glycine was added with strong recovery rates noted especially in the area of negative and cognitive symptoms. Glycine, however, sporadic results aside (dose 60 g/day or 0.8 g/kg, [ 2 ] [ 3 ]  approximately the amount in 300 g of gelatin powder or two kilograms of sunflower seeds [ 4 ] ) remains an adjunct  antipsychotic  and an unworkable compound. However, the  Eli Lilly and Company  study drug  LY-2140023  is being studied as a primary  antipsychotic  and is showing strong recovery rates, especially in the area of negative and cognitive symptoms of  schizophrenia .  Tardive dyskinesia ,  diabetes  and other standard complications have not been noted:


Treatment with LY2140023, like treatment with olanzapine, was safe and well-tolerated; treated patients showed statistically significant improvements in both positive and negative symptoms of schizophrenia compared to placebo (P = 0.001 at week 4). Notably, patients treated with LY-2140023 did not differ from placebo-treated patients with respect to prolactin elevation, extrapyramidal symptoms or weight gain. These data suggest that mGlu2/3 receptor agonists have antipsychotic properties and may provide a new alternative for the treatment of schizophrenia. [ 5 ]


Other NMDA receptor modulators are being studied and this modality of treatment may once approved as  antipsychotic  medications gradually replace the current (dopaminergic) antipsychotics.


NNC2215  is a  bioengineered  glucose-sensitive  insulin . The drug is designed by a team of  Novo Nordisk  researchers. [ 1 ]


NNC2215 can sense the glucose concentration presence in blood. The protein's sensitivity is reduced when low concentration of glucose, thus reducing the risk of  hypoglycemia . [ 2 ]  In addition, it can effectively cover the risk of fluctuations of blood sugar levels.


The study was published on scientific journal  Nature  on October 16, 2024. [ 3 ]  This study demonstrated the ability of protein engineering in future medicine and a major advancement in treatment capabilities.


A glucose sensitive treatment method for  diabetes  have long been pursued by science since 1979. [ 4 ]  Such protein is expected to solve the problem of  fluctuations of blood sugar levels .  For diabetes patients, skipping meal alone could lead to  hypoglycemia , which is a common complication could cause  loss of consciousness  or  seizures . [ 5 ]


There are several attempts to create such a medicine with various levels of success. [ 6 ] [ 7 ]


Nomeco , a wholly owned subsidiary of  Phoenix Group , is the largest wholesaler of pharmaceutical products in  Denmark . The company is headquartered in  Copenhagen  and is building a 20,000 square metre Nordic logistics centre in  Køge  which is expected to open in 2017. [ 2 ]


Nomeco was founded in 1991, through the merger of Mecobenzon A/S (founded 1919),  and Nordisk Droge og Kemikalie A/S (founded 1903). In 1998, the company was merged with  Finnish  Tamro Oyj, the largest wholesaler of pharmaceutical products in the  Nordic  and  Baltic countries . In 2000,  Phoenix Pharmahandel  became the largest shareholder of the company with 33.7% of the shares and in 2005, the company acquired full ownership. In August 2015, it was announced that Nomeco will build a new Nordic logistics centre for pharmaceuticals in  Køge  south of Copenhagen. [ 3 ]


Nomeco is headquartered in the  South Harbour  area of Copenhagen. The new logistics centre for pharmaceuticals in Køge will cover 20,000 square metres and be located in Skandinavisk Transport Center. Will open in the summer of 2018. [ 3 ]


The company is Denmark's largest distributor of  pharmaceuticals  and other products that promote health and well-being. Nomeco's wide range of services include various information logistics, marketing, sales and registration expert services. Pharmaceutical distribution is Nomeco's core business. Nomeco's customers are pharmaceutical companies, pharmacies, hospitals and retailers.


This Danish corporation or company article is a  stub . You can help Wikipedia by  expanding it .


NONMEM  is a  non-linear mixed-effects modeling software  package developed by Stuart L. Beal and Lewis B. Sheiner in the late 1970s at  University of California, San Francisco , and expanded by Robert Bauer at Icon PLC. Its name is an acronym for  nonlinear mixed effects modeling  but it is especially powerful in the context of  population pharmacokinetics ,  pharmacometrics , and  PK/PD models . [ 1 ] [ 2 ] [ 3 ] [ 4 ]
NONMEM models are written in NMTRAN, a dedicated  model specification language  that is translated into  FORTRAN , compiled on the fly and executed by a command-line script. Results are presented as text output files including tables. [ 5 ]   There are multiple interfaces to assist modelers with housekeeping of files, tracking of model development, goodness-of-fit evaluations and graphical output, such as PsN and xpose [ 6 ]  and Wings for NONMEM. [ 7 ]  Current version for NONMEM is 7.5.


NONMEM estimates its models according to principles of  maximum likelihood estimation .  nonlinear mixed-effects model  generally do not have close-formed solutions, and therefore specific estimation methods are applied, such as linearization methods as first-order (FO), first-order conditional (FOCE) or the laplacian (LAPL), approximation methods such as iterative-two stage (ITS), importance sampling (IMP), stochastic approximation estimation (SAEM) or direct sampling. [ 8 ]


A  nonsteroidal  compound is a drug that is not a  steroid  nor a steroid derivative. [ 1 ] [ 2 ]   Nonsteroidal anti-inflammatory drugs  (NSAIDs) are distinguished from  corticosteroids  as a class of  anti-inflammatory agents . [ 3 ]


Examples include the following: [ 1 ] [ 2 ]


19-Noretiocholanolone , also known as  5β-estran-3α-ol-17-one , is a  metabolite  of  nandrolone  (19-nortestosterone) and  bolandione  (19-norandrostenedione) that is formed by  5α-reductase . It is on the list of substances prohibited by the  World Anti-Doping Agency  since it is a detectable metabolite of nandrolone, an  anabolic-androgenic steroid  (AAS). [ 1 ]


Consumption of boar meat, liver, kidneys and heart have been found to increase urinary 19-noretiocholanolone output. [ 2 ]


Hospital Pharmacy of North Norway Trust  ( Norwegian :  Sykehusapotek Nord HF ,  Northern Sami :  Davvi Buohcceviessoapotehka ) is a  health trust  owned by  Northern Norway Regional Health Authority  that operates six  hospital pharmacies . The pharmacies are part of the  Ditt Apotek  chain and use  Norsk Medisinaldepot  as  wholesaler .


The pharmacies are located at  Tromsø University Hospital ,  Bodø Hospital ,  Harstad Hospital ,  Narvik Hospital ,  Lofoten Hospital  and  Stokmarknes Hospital .


The  Norwegian Medical Products Agency  formerly the  Norwegian Medicines Agency  ( Direktoratet for Medisinske Produkter  tidl.  Statens legemiddelverk ,  Legemiddelverket ) is the national regulatory authority in  Norway  for new and existing  pharmaceuticals ,  medical devices  and for the  supply chain  security of these. The Agency is responsible for supervising the production,  trials  and  marketing  of medicines. It approves medicines and monitors their use, and ensures cost-efficient, effective and well-documented use of medicines. The Inspectorate also supervises the supply chain and regulates pharmacy prices and trade conditions. [ 1 ]


Norwegian Pharmacy Association  ( Norwegian :  Apotekforeningen ) is a  trade organization  representing the  pharmacies  and pharmacy owners in  Norway . In addition to the three large chains and hospital pharmacies, 20 independent pharmacists are members of the organization.


The Norwegian Pharmacy Association is a trade association for Pharmacies located in Norway. It was established in 1881. [ 1 ]


Notable people include  Leif Brendel , secretary-general from 1940 to 1968.


This article about an organisation based in Norway is a  stub . You can help Wikipedia by  expanding it .


Novolin  is the  brand name  of three distinct insulin-containing products [ 1 ]  manufactured by  Novo Nordisk : [ 2 ]


NPS-2143  ( SB-262,470A ) is a  calcilytic  drug which acts as an  antagonist  at the  Calcium-sensing receptor  (CaSR), and consequently stimulates release of  parathyroid hormone . [ 1 ]  Calcilytic drugs have been researched as potential treatments for  osteoporosis , and as the first such compound developed, NPS-2143 is still widely used in research into the CaSR receptor as well as design of newer calcilytic agents. [ 2 ] [ 3 ] [ 4 ]


NRICM101  ( Chinese :  清冠一號 ; Taiwan: Chingguan Yihau; Mainland: Qīng-guān Yī-hào), is a treatment for  COVID-19  developed in  Taiwan  using  Traditional Chinese Medicine  (TCM), created by the  National Research Institute of Chinese Medicine  (NRICM), a governmental body of Taiwan. [ 1 ]  The prescription has gained legal approval in several countries, the first TCM recipe to do so for COVID-19 treatment. [ 2 ]  In its native Taiwan, the treatment is used in conjunction with vaccinations. [ 3 ]  Amongst the formulations available are  RespireAid  from  Sun Ten (順天堂)  [ zh ] , and  COVRelief  from  Chuang Song Zong (莊松榮)  [ zh ] . [ 4 ] [ 5 ]


NRICM101 contains 10  herbal medicinal components , including  indigowoad root ,  fish mint ,  Chinese skullcap ,  houttuynia , and  licorice . [ 4 ] [ 6 ] [ 7 ]


According to the NRICM, the herbal medicine is supposed to work by preventing the  SARS-CoV2  virus from binding with ACE2. [ 7 ]  This would reduce the chance of severe illness. [ 5 ]


On 2021 May 18, Taiwan licensed NRICM101 for distribution under emergency access policy. It became available as  dietary supplement  in the  U.S ., the  UK ,  Australia , the EU. [ 4 ] [ 5 ]


This  Taiwan -related article is a  stub . You can help Wikipedia by  expanding it .


This  China -related article is a  stub . You can help Wikipedia by  expanding it .


NS-136  is a  selective   muscarinic acetylcholine   M 4  receptor   positive allosteric modulator  which is under development for the treatment of  schizophrenia  and  Alzheimer's disease . [ 1 ] [ 3 ] [ 2 ]  It has been found to possess  pro-cognitive  effects in rodents. [ 2 ]  The drug is under development by NeuShen Therapeutics. [ 1 ] [ 3 ]  As of May 2024, it is in  phase 1   clinical trials  for schizophrenia and is in the  preclinical stage of development  for Alzheimer's disease. [ 1 ] [ 3 ]  The drug is a  small molecule , but its  chemical structure  does not seem to have been disclosed. [ 1 ] [ 3 ]


Nufenoxole  ( SC-27166 ) is an  antidiarrhoeal  drug which acts as a peripherally selective  opioid  agonist, in a similar manner to  loperamide . while it is able to activate  μ-opioid receptors , it fails to cross the  blood–brain barrier  and so has a selective action against diarrhoea without producing  analgesic  effects. [ 1 ] [ 2 ] [ 3 ] [ 4 ] [ 5 ]


NV-5440  is a drug which acts as both a non-specific inhibitor of the  glucose transporters  and also a selective inhibitor of  mTORC1 , with no significant action at the related  mTORC2  subtype. Compounds of this type have potential application in the treatment of  cancer , and it is also used for research into the links between  calorie restriction  and  longevity . [ 1 ] [ 2 ] [ 3 ]


O-4310  ( 1-isopropyl-6-fluoro-psilocin ) is a  tryptamine   derivative  developed by  Organix Inc  which acts as a  serotonin receptor   agonist . It is claimed to have an EC 50  of 5   nM at the  5-HT 2A  receptor  with 89%  efficacy  relative to  serotonin , and 100-fold  selectivity  over the  5-HT 2C  receptor , while being apparently inactive at the  5-HT 2B   antitarget . [ 1 ] [ 2 ]


Obecabtagene autoleucel , sold under the brand name  Aucatzyl , is an  anti-cancer medication  used for the treatment of  acute lymphoblastic leukemia . [ 1 ] [ 2 ]  It is a  CD19 -directed genetically modified autologous  T-cell  immunotherapy. [ 1 ] [ 2 ]


The most common side effects include  cytokine release syndrome , infections-pathogen unspecified, musculoskeletal pain, viral infections, fever, nausea, bacterial infectious disorders, diarrhea, febrile neutropenia, immune effector cell-associated  neurotoxicity syndrome , hypotension, pain, fatigue, headache, encephalopathy, and hemorrhage. [ 2 ]


Obecabtagene autoleucel was approved for medical use in the United States in November 2024. [ 2 ] [ 3 ]


Obecabtagene autoleucel is  indicated  for the treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia. [ 1 ] [ 2 ]


The US  Food and Drug Administration  (FDA) approved prescribing information for obecabtagene autoleucel has a  boxed warning  for  cytokine release syndrome , immune effector cell-associated  neurotoxicity syndrome , and  T-cell  malignancies. [ 2 ]


The most common side effects include cytokine release syndrome, infections-pathogen unspecified, musculoskeletal pain, viral infections, fever, nausea, bacterial infectious disorders, diarrhea, febrile neutropenia, immune effector cell-associated neurotoxicity syndrome, hypotension, pain, fatigue, headache, encephalopathy, and hemorrhage. [ 2 ]


Efficacy was evaluated in FELIX (NCT04404660), an open-label, multicenter, single-arm trial that enrolled adults with relapsed or refractory CD19-positive B-cell acute lymphoblastic leukemia. [ 2 ]  Enrolled participants were required to have relapsed following a remission lasting twelve months or less, relapsed or refractory acute lymphoblastic leukemia following two or more prior lines of systemic therapy, or disease that was relapsed or refractory three or more months after allogeneic stem cell transplantation. [ 2 ] [ 4 ]


The major efficacy outcome measures were rate and duration of complete remission achieved within three months after infusion. [ 2 ]  Additional outcome measures were rate and duration of overall complete remission which includes complete remission and complete remission with incomplete hematologic recovery, at any time. [ 2 ]  Of the 65 participants evaluable for efficacy, 27 participants (42%; 95% confidence interval [CI]: 29%, 54%) achieved complete remission within three months. [ 2 ]  The median duration of complete remission achieved within three months was 14.1 months (95% CI: 6.1, not reached). [ 2 ]


The US  Food and Drug Administration  (FDA) granted the application for obecabtagene autoleucel regenerative medicine advanced therapy (RMAT) and  orphan drug  designations.


Obecabtagene autoleucel was approved for medical use in the United States in November 2024. [ 2 ]


Obecabtagene autoleucel is the  international nonproprietary name . [ 5 ]


It is sold under the brand name Aucatzyl. [ 1 ] [ 2 ]


Obefazimod  is an  investigational new drug  that is being evaluated for the treatment of  ulcerative colitis . [ 1 ]   It is  miR-124  micro RNA upregulator. [ 2 ]


Obexelimab  is an experimental drug developed to treat  IgG4-related disease  and  lupus . It works as a "bifunctional, non-cytolytic, humanised  monoclonal antibody  that binds  CD19  and  Fc gamma receptor IIb  to inhibit  B cells ,  plasmablasts , and CD19-expressing  plasma cells ." [ 1 ] [ 2 ]


Ocedurenone , formerly known as  KBP-5074 , [ 1 ]  is a nonsteroidal, selective  mineralocorticoid receptor antagonist  that is being developed to treat  hypertension  in patients with  chronic kidney disease  with less risk of  hyperkalemia  than existing treatments. [ 2 ] [ 3 ]  In 2023, KPB Biosciences entered into talks to sell the drug to  Novo Nordisk  for USD$1.3 billion. It is a  small molecule  drug administered orally and is in a Phase III trial that is scheduled to complete in 2024. [ 4 ] [ 5 ]


Ocrelizumab/hyaluronidase , sold under the brand name  Ocrevus Zunovo , is a  fixed-dose combination  medication used for the treatment of  multiple sclerosis . [ 1 ]  It contains  ocrelizumab , a recombinant humanized  monoclonal antibody  directed at CD20; [ 1 ]  and  hyaluronidase  (human recombinant), an  endoglycosidase . [ 1 ]  It is taken by  subcutaneous injection . [ 1 ]


Ocrelizumab/hyaluronidase was approved for medical use in the United States in September 2024. [ 2 ] [ 3 ]


Ocrelizumab/hyaluronidase is  indicated  for the treatment of relapsing forms of multiple sclerosis, to include clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease; [ 1 ]  and primary progressive multiple sclerosis. [ 1 ]


Odilorhabdins  are a class of natural  antibacterial agents  produced by the bacterium  Xenorhabdus nematophila . [ 1 ]  Odilorhabdins act against both  Gram-positive  and  Gram-negative  pathogens, and were shown to eliminate infections in mouse models. [ 1 ]


Odilorhabdins interfere with the pathogen's  protein synthesis  and are  ribosome -targeting. They bind to the small ribosomal subunit at a site not exploited by previous antibiotics and induce miscoding and premature  stop codon  bypass. [ 1 ]  Odilorhabdins were shown to act particularly against  carbapenem -resistant members of bacteria family  Enterobacteriaceae , having potential to kill pathogens with  antimicrobial resistance . [ 1 ]


The discovery of odilorhabdins was announced in 2013 [ 2 ]  and formally described in 2018 by the researchers of the  University of Illinois at Chicago  and Nosopharm. To identify the antibiotic, the Nosopharm researchers tested 80  cultured  bacterial  strains  for antimicrobial properties and then isolated the active compounds. [ 3 ]


Odronextamab , sold under the brand name  Ordspono , is a  CD20  x  CD3  bispecific  monoclonal antibody  that is used for the treatment of  follicular lymphoma  or  diffuse large B-cell lymphoma . [ 1 ] [ 2 ]  It was developed by  Regeneron Pharmaceuticals . [ 1 ]


The most common side effects include  cytokine release syndrome ,  infections ,  neutropenia ,  pyrexia  (fever),  anemia ,  thrombocytopenia , and  diarrhea . [ 1 ]


Odronextamab was approved for medical use in the European Union in August 2024. [ 1 ] [ 2 ]


Odronextamab is  indicated  for the treatment of adults with relapsed or refractory follicular lymphoma after two or more lines of systemic therapy; [ 1 ]  and for the treatment of adults with relapsed or refractory diffuse large B‑cell lymphoma after two or more lines of systemic therapy. [ 1 ]


The most common side effects include cytokine release syndrome, infections, neutropenia, pyrexia, anemia, thrombocytopenia, and diarrhea. [ 1 ]


In June 2024, the  Committee for Medicinal Products for Human Use  of the  European Medicines Agency  adopted a positive opinion, recommending the granting of a conditional marketing authorization for the medicinal product Ordspono, intended for the treatment of follicular lymphoma and diffuse large B-cell lymphoma. [ 1 ] [ 3 ]  The applicant for this medicinal product is Regeneron Ireland Designated Activity Company. [ 1 ]  Odronextamab was approved for medical use in the European Union in August 2024. [ 1 ] [ 2 ] [ 4 ]


Odronextamab is the  international nonproprietary name . [ 5 ]


Oenethyl , also known as  2-methylaminoheptane  and sold under the brand names  Pacamine  and  Neosupranol , is a  sympathomimetic  and  vasopressor   medication  of the  alkylamine  which is no longer marketed. [ 1 ] [ 2 ] [ 3 ]  It was used as a  nasal decongestant  and to control  blood pressure  during  anesthesia . [ 3 ]  It is closely  structurally related  to other alkylamines, for instance  methylhexanamine  and  tuaminoheptane , among others. [ 3 ]  These compounds are known to act as structurally simple  monoamine releasing agents  and to produce  psychostimulant -like effects. [ 4 ] [ 5 ] [ 6 ] [ 2 ]


Olezarsen , sold under the brand name  Tryngolza , is a  medication  used in the treatment of  familial chylomicronemia syndrome . [ 1 ] [ 2 ]  Olezarsen is an  apolipoprotein C-III -directed  antisense oligonucleotide . [ 1 ]  It is given by  injection under the skin . [ 1 ]


Olezarsen was approved for medical use in the United States in December 2024. [ 1 ] [ 3 ]


Olezarsen is  indicated  as an adjunct to diet to reduce triglycerides in adults with  familial chylomicronemia syndrome . [ 1 ]


The US  Food and Drug Administration  (FDA) granted the application of olezarsen  orphan drug  designation in February 2024. [ 4 ]


Olezarsen was approved for medical use in the United States in December 2024. [ 3 ] [ 5 ]


Olezarsen is the  international nonproprietary name . [ 6 ]


Olezarsen is sold under the brand name Tryngolza. [ 1 ]


Olpasiran  (AMG890) is an experimental  antisense therapy  designed to lower the level of  lipoprotein(a) , which is believed to be a causal factor in the development of  cardiovascular disease . The drug is developed by  Amgen . [ 1 ] [ 2 ]


Olutasidenib , sold under the brand name  Rezlidhia , is an  anticancer medication  used to treat relapsed or refractory  acute myeloid leukemia  with a susceptible IDH1 mutation. [ 2 ] [ 3 ]  Olutasidenib is an  isocitrate dehydrogenase-1  (IDH1) inhibitor. [ 2 ]  It is taken  by mouth . [ 2 ]


The most common adverse reactions include nausea, fatigue/malaise, arthralgia, constipation, leukocytosis, dyspnea, fever, rash, mucositis, diarrhea, and transaminitis. [ 4 ]


Olutasidenib was approved for medical use in the United States in December 2022, [ 2 ] [ 3 ] [ 4 ] [ 5 ] [ 6 ] [ 7 ]  based on the phase 1 results of a phase 1/2 trial. [ 8 ]


Olutasidenib is indicated for the treatment of adults with relapsed or refractory acute myeloid leukemia with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test. [ 2 ] [ 3 ] [ 4 ]


Olutasidenib is the international nonproprietary name. [ 9 ]


Omidubicel , sold under the brand name  Omisirge , is a blood-based  cell therapy  used for the treatment of  blood cancers . [ 3 ] [ 4 ] [ 5 ]  Omidubicel is a  nicotinamide -modified allogeneic hematopoietic progenitor cell therapy derived from  cord blood . [ 3 ]


The most common adverse reactions include infections,  graft-versus-host disease , and infusion reactions. [ 3 ]


Omidubicel is composed of human allogeneic stem cells from umbilical cord blood that are processed and cultured with nicotinamide (a form of vitamin B3). [ 3 ]  Each dose is patient-specific, containing healthy stem cells from an allogeneic pre-screened donor, meaning it comes from a different individual rather than using the patient's own cells. [ 3 ]  Omidubicel was approved for medical use in the United States in April 2023. [ 3 ] [ 6 ]


Omidubicel is  indicated  for use in people twelve years of age and older with blood cancers (hematologic malignancies) planned for umbilical cord blood  transplantation  following myeloablative conditioning to reduce the time to  neutrophil  recovery and the incidence of infection. [ 3 ] [ 6 ]


The US  Food and Drug Administration  (FDA) evaluated the safety and effectiveness of omidubicel based on a randomized, multicenter study comparing transplantation of omidubicel to transplantation of umbilical cord blood, in subjects between the ages of 12 and 65 years. [ 3 ]  The study enrolled a total of 125 participants. [ 3 ]  All participants in the study had confirmed blood cancers. [ 3 ]  The efficacy of omidubicel was based on the amount of time needed for recovery of the participant's neutrophils (a type of white blood cell that helps protect the body from infections) and the incidence of infections following transplantation. [ 3 ]  In total, 125 participants were randomized--62 participants to receive omidubicel-onlv and 63 to the unmanipulated cord blood group. [ 6 ]  Fifty-two participants were transplanted with omidubicel-onlv receiving a median CD34+ cell dose of 9.0 X 106 cells/kg (range 2.1 – 47.6 X 106 cells/kg). [ 6 ]  Fifty-six participants were transplanted in the unmanipulated cord blood arm with one or two cord units (66% received two cord units). [ 6 ]  In the 42 participants with reported post-thaw cell dose, the median CD34+ cell dose was 0.2 X 106 cells/kg (range 0.0 – 0.8 X 106 cells/kg). Multiple conditioning regimens were used, including total body irradiation-based or chemotherapy-based options. [ 6 ]


The FDA granted the application for omidubicel  priority review ,  breakthrough therapy , and  orphan drug  designations. [ 3 ] [ 6 ]  The FDA granted regular approval of Omisirge to Gamida Cell Ltd. [ 3 ]


Omzotirome  ( INN ), formerly codenamed  TRC-150094 , is a  thyromimetic  drug which acts as a metabolic modulator which restores  metabolic flexibility . It has been shown to improve  insulin resistance  and  hyperglycemia , and is in Phase III human  clinical trials  for the treatment of Cardiometabolic-Based Chronic Disease (CMBCD) by improving  dysglycemia , dyslipidemia and hypertension. [ 1 ] [ 2 ] [ 3 ] [ 4 ] [ 5 ]


Onchidal  is a naturally occurring  neurotoxin  produced as a defensive secretion by the  mollusc   Onchidella binneyi  and several other related species in  Onchidella ,  a genus of small, air-breathing sea slugs. It acts as an irreversible  acetylcholinesterase inhibitor , the same mechanism of action as that of the deadly  nerve agents , however onchidal is not an  organophosphorus  or  carbamate  compound and bears little resemblance to other compounds of this nature. [ 1 ]


The  Oncologic Drugs Advisory Committee  (ODAC) receives requests for technical and clinical evaluation of new drugs by the  U.S. Food and Drug Administration  (FDA).  The committee, consisting of members from academic and clinical oncology biostatistics, the general public, and the pharmaceutical industry, makes non-binding recommendations to both the  CDER  and  CBER  divisions of the FDA about the advisability of approving new medications to treat  cancer .


This  oncology  article is a  stub . You can help Wikipedia by  expanding it .


Onfasprodil  ( MIJ821 ) is a drug delivered via  intravenous infusion  that is designed as a fast-acting treatment for  treatment-resistant depression . It works as a  negative allosteric modulator  of the  NMDA receptor   subunit 2B  (NR2B). The drug is developed by  Novartis . [ 1 ] [ 2 ]


Onset of action  is the duration of time it takes for a  drug 's effects to come to prominence upon administration. With  oral administration , it typically ranges anywhere from 20 minutes to over an hour, depending on the drug in question. Other methods of ingestion such as  smoking  or  injection  can take as little as seconds to minutes to take effect. The determination of the onset of action, however, is not completely dependent upon  route of administration . There are several other factors that determine the onset of action for a specific drug, including  drug formulation ,  dosage , and the patient receiving the drug.


A drug's pharmacological effects can only occur once it has been fully solubilized and has entered the blood stream. For most drugs administered orally, the drug must be ingested, pass through the stomach, and into the small intestine, where the drug molecules enter the blood stream through the  villi  and  microvilli . [ 1 ]  A few drugs such as alcohol are absorbed by the lining of the stomach, and therefore tend to take effect much more quickly than the vast majority of oral medications which are absorbed in the small intestine. Gastric emptying time can vary from 0 to 3 hours, [ 2 ]  and therefore plays a major role in onset of action for orally administered drugs. For intravenous administration, the pathway is much shorter because the drug is administered (usually already in solution) directly to the bloodstream.


OPC-64005  is a  serotonin–norepinephrine–dopamine reuptake inhibitor  (SNDRI), or "triple reuptake inhibitor" (TRI), which is under development for the treatment of  major depressive disorder . [ 1 ] [ 2 ] [ 3 ]  It was also under development for the treatment of  attention deficit hyperactivity disorder  (ADHD), but development for this indication was discontinued. [ 1 ] [ 4 ]  It is taken  by mouth . [ 1 ]


As of December 2022, OPC-64005 is in  phase 2   clinical trials  for major depressive disorder. [ 1 ] [ 2 ]  It reached phase 2 clinical trials for ADHD prior to the discontinuation of its development for this use. [ 1 ]  It completed a phase 2 clinical trial for ADHD comparing it with  placebo  and  atomoxetine , but the results of this trial were not disclosed. [ 5 ]  The drug is under development by  Otsuka Pharmaceutical . [ 1 ] [ 2 ]  It is a  small molecule , but its  chemical structure  does not appear to have been disclosed. [ 1 ] [ 2 ]


Opevesostat  is an  investigational new drug  being developed for the treatment of  metastatic castration-resistant prostate cancer  (mCRPC). [ 1 ]  It is a non-steroidal, selective inhibitor of  CYP11A1  (cholesterol side-chain cleavage enzyme) [ 2 ]  discovered by  Orion Corporation  and currently undergoing clinical development by  Merck & Co., Inc.  Opevesostat's mechanism of action involves suppressing the production of steroid hormones and their precursors that may activate the  androgen receptor  signaling pathway, which is crucial in prostate cancer progression. As of 2024, opevesostat is being evaluated in two Phase 3 clinical trials, OMAHA1 and OMAHA2a, which are assessing its efficacy and safety in combination with hormone replacement therapy for patients with mCRPC who have failed prior treatments. [ 3 ] [ 4 ] [ 5 ]


Opnurasib  (JDQ-443) is a  small-molecule  covalent  KRASG12C  inhibitor developed for  non-small-cell lung cancer . [ 1 ] [ 2 ] [ 3 ] [ 4 ]


Opodeldoc  is a medical plaster or  liniment  invented, or at least named, by the  German Renaissance   physician   Paracelsus  in the 1500s.  In modern form opodeldoc is a mixture of  soap  in  alcohol , to which  camphor  and sometimes a number of  herbal  essences, most notably  wormwood , are added.


In his  Bertheonea Sive Chirurgia Minor  published in 1603, Paracelsus mentioned "oppodeltoch" twice, but with uncertain ingredients. [ 1 ]


As to the origin of the name,  Kurt Peters  speculated that it was coined by Paracelsus from syllables from the words " opo ponax, b del lium, and aristol och ia."  Opoponax  is a variety of  myrrh ;  bdellium  is  Commiphora wightii , which produces a similar resin; and  Aristolochia  is a widely distributed  genus  which includes  A. pfeiferi ,  A. rugosa  and  A.  trilobata  that are used in folk medicine to cure snakebites. The name suggests that these aromatic plants may have figured in Paracelsus's recipe. [ 2 ]


In his  Medicina Militaris  of 1620, German military physician Raymund Minderer ("Mindererus"; 1570-1621) praised the Paracelsus compound as a plaster, good for wounds. Minderer compared it to his own variant, which set more like sealing wax. [ 3 ]  Opodeldoc and Paracelsus were acknowledged in English no later than 1646, in  Sir Thomas Browne 's popular and influential  Pseudodoxia Epidemica . [ 4 ]


Paracelsus's recipe is completely unrelated to later preparations of the same name. [ 5 ]  By the second printing of the  Edinburgh Pharmacopoeia  in 1722 the name applied to a soap-based liniment. Such a liniment in patent form, sold by  John Newbery 's company in Great Britain "ever since A.D. 1786", was called "Dr. Steer's Opodeldoc". [ 6 ]  Produced for decades, the "Dr. Steer" preparation had been successfully imported into the U.S., and was common enough there to rank as one of the eight patent medicines to be analyzed (although not condemned) by the  Philadelphia College of Pharmacy  in 1824. [ 7 ]


The name  Old Opodeldoc  was formerly used as a standard name for a  stock character  who was a physician, especially when played as a  comic  figure.  Edgar Allan Poe  used "Oppodeldoc" as a  pseudonym  for a character in the  short story  " The Literary Life of Thingum Bob, Esq. " [ 2 ]


The  Pharmacopoeia  of the  United States  (U.S.P.) gives a recipe for opodeldoc that contains:


As late as the early 1990s 'Epideldoc' (sic) was compounded on request by several pharmacists in the Northwest of England.


Ordesekimab  is an anti- interleukin 15   monoclonal antibody  developed by  Amgen  to treat refractory  celiac disease . [ 1 ] [ 2 ] [ 3 ] [ 4 ]


Oritinib  is an  investigational new drug  currently under investigation for its potential use in cancer treatment. [ 1 ] [ 2 ]  As a  epidermal growth factor receptor  (EGFR)  tyrosine kinase inhibitor , oritinib targets specific enzymes involved in the signaling pathways that regulate cell division and survival, which are often dysregulated in cancer cells. [ 1 ]


Ortho-McNeil Pharmaceutical  (now operating under  Janssen Pharmaceuticals ) was a pharmaceutical company based in Raritan, New Jersey, that was formed from the merger of  Ortho Pharmaceutical  and  McNeil Pharmaceutical  in 1993. [ 1 ]  Both of these pharmaceutical companies were pioneers and leaders in areas such as  pain management ,  acid reflux disease , and  infectious diseases . [ 2 ]


Ortho-McNeil and  Janssen Pharmaceuticals  together composed the  Ortho-McNeil-Janssen  group within  Johnson & Johnson  prior to a decision to operate under the Janssen Pharmaceuticals name in 2011. [ 3 ]


Amongst its many  prescription drugs  are:


In 2010, Ortho-McNeil pled guilty in U.S. District Court to one count of misdemeanor violation of the  Food, Drug & Cosmetic Act  for illegally promoting its epilepsy drug  Topamax  for uses that were not approved by the FDA. The company was charged with using a program called "Doctor for a Day" to promote Topamax to psychiatrists for treatment of mental health conditions, despite never applying for FDA approval of Topimax for any psychiatric indication. The company was sentenced to pay a fine of $6.14 million. [ 4 ]


Ortho-McNeil's parent company, Johnson and Johnson, also paid $75.37 million to resolve civil allegations under the  False Claims Act  that it caused false claims to be submitted to government health care programs for a variety of psychiatric uses that were not FDA approved. [ 4 ]


Ortho-McNeil was found liable in two 2013 civil suits by women who gave birth to children with birth defects after taking Topamax while pregnant. The jury found that they negligently failed to warn the patients and their doctors of the risks associated with Topamax when used by patients during pregnancy. [ 5 ]  They awarded $11 million in damages to one family [ 6 ]  and $4 million to the other. [ 5 ]


As a result of these and other patient reports, the FDA ordered that a warning be added to the prescribing information for Topamax detailing the risk of birth defects such as cleft lip and cleft palate. [ 5 ]


Oryzalin  is a  herbicide  of the  dinitroaniline  class.  It acts through the disruption ( depolymerization ) of microtubules, thus blocking  anisotropic  growth of plant cells. [ 1 ]  It can also be used to induce  polyploidy  in plants as an alternative to  colchicine . [ 2 ]


Osoresnontrine  ( BI-409306 ) is a  phosphodiesterase 9 inhibitor  in development for  schizophrenia , [ 1 ]   attenuated psychosis syndrome , [ 2 ]  and  Alzheimer's disease . [ 3 ]  A preclinical study suggested that it increases memory in rodents. [ 4 ]


OUP-16  is a  histamine agonist  selective for the  H 4  subtype. [ 1 ]


Oxomemazine/guaifenesin  is a  sedative   antihistamine  plus  expectorant . [ 1 ] [ 2 ]   It is a combination of  oxomemazine  and  guaifenesin .


It is sold under a variety of brand names including  Toplexil  by  Aventis . It is sold over the counter in  France ,  Morocco ,  Egypt , Algeria,  South Korea  and the  UAE [ citation needed ] . The  oxomemazine  ingredient (antihistamine) is not approved by the U.S.  Food and Drug Administration  (FDA), either for over the counter or prescription use. It is sold in France as cough syrup. French travelers also use it as a sleeping aid for long flights. [ citation needed ]


Padeliporfin , sold under the brand name  Tookad , is a  medication  used to treat men with  prostate cancer . [ 1 ] [ 2 ]  It is used in the form padeliporfin di-potassium. [ 2 ]


The most common side effects include problems with urinating (pain, inability to pass urine, strong urge to pass urine, frequent urination, and incontinence), sexual problems (erectile dysfunction and ejaculation failure), blood in urine, urinary tract infection, and pain and bleeding around the genital area. [ 2 ]


Padeliporfin was approved for medical use in the European Union in November 2017. [ 2 ]


Padeliporfin is  indicated  as monotherapy for adults with previously untreated, unilateral, low risk,  adenocarcinoma  of the  prostate  with a life expectancy of at least ten years and clinical stage  T1c  or  T2a ;  Gleason score  ≤ 6, based on high-resolution biopsy strategies;  prostate-specific antigen  (PSA) ≤ 10   ng/mL; three positive cancer cores with a maximum cancer core length of 5   mm in any one core or 1-2 positive cancer cores with ≥ 50% cancer involvement in any one core or a PSA density ≥ 0.15   ng/mL/cm 3 . [ 2 ]


Padeliporfin was approved for use in the European Union in November 2017. [ 2 ]


In February 2020, the oncologic drugs advisory committee of the US  Food and Drug Administration  (FDA) voted against approving padeliporfin di-potassium powder for solution for injection, submitted by Steba Biotech, S.A. The proposed indication (use) for the product is for the treatment of men with localized prostate cancer, meeting the following criteria: Stage T1-T2a and prostate specific antigen less than or equal to 10 ng/mL and Gleason Grade Group 1 based on transrectal ultrasound guided biopsy or unilateral Gleason Grade Group 2 based on multiparametric magnetic resonance imaging-targeted biopsy with less than 50 percent of cores positive. [ 3 ] [ 4 ]


Paediatric-use marketing authorisations  ( PUMA ) are granted by the  European Medicines Agency  (EMA) for medical products that are intended exclusively for  paediatric  use, that is, for use in patients younger than 18 years. Like ordinary EMA marketing authorisations, a PUMA approval is valid in all countries of the  European Economic Area  (the  European Union  as well as  Iceland ,  Liechtenstein , and Norway). The PUMA process was established to make it more profitable for pharmaceutical companies to market drugs for children. For this purpose, new data used for PUMA approved drugs are protected for 10 years, and the applications are partially exempt from fees. [ 1 ]


In September 2011, the first drug was approved under this process. It was Buccolam, a  buccal  application form of  midazolam  for the treatment of  seizures . [ 2 ] [ 3 ]


Palazestrant  is an  investigational new drug  which is being evaluated for the treatment of estrogen receptor-positive (ER+)  breast cancer , with a dual mechanism of action as both a complete  estrogen receptor antagonist  (CERAN) and a  selective estrogen receptor degrader  (SERD). This orally bioavailable small molecule has demonstrated potent activity against both wild-type and mutant forms of the estrogen receptor. [ 1 ]


Paltusotine  is a selective  somatostatin receptor  type 2 (SST2) agonist in development for the treatment of  acromegaly  and certain neuroendocrine tumors. It is a  small molecule  delivered orally. [ 1 ] [ 2 ] [ 3 ] [ 4 ]


Pamapimod  is an  investigational drug  which is being evaluated for the treatment of  autoimmune diseases . It is a  p38 mitogen-activated protein kinase  inhibitor. [ 1 ]  It has been evaluated in a phase 2 clinical trial for the treatment of  rheumatoid arthritis , but was found not to be effective. [ 2 ] [ 3 ]  It has subsequently been investigated as a possible treatment for  osteoarthritis . [ 4 ] [ 5 ]


Pan-assay interference compounds  ( PAINS ) are  chemical compounds  that often give  false positive  results in  high-throughput screens . [ 1 ]  PAINS tend to  react  nonspecifically with numerous  biological targets  rather than specifically affecting one desired target. [ 2 ]  A number of disruptive  functional groups  are shared by many PAINS. [ 2 ] [ 3 ] [ 4 ]


While a number of filters have been proposed and are used in  virtual screening  and  computer-aided drug design , [ 5 ]  the accuracy of filters with regard to compounds they flag and don't flag has been criticized. [ 6 ]


Common PAINS include  toxoflavin ,  isothiazolones ,  hydroxyphenyl hydrazones ,  curcumin ,  phenol-sulfonamides ,  rhodanines ,  enones ,  quinones , and  catechols . [ 7 ]


Pantopon , also known as Opium Alkaloids Hydrochlorides, is a preparation of  opiates  made up of all of the  alkaloids  present in  opium  in their natural proportions as  hydrochlorides   salts . [ 1 ] [ 2 ] [ 3 ]  It can sometimes be tolerated by people who are allergic to  morphine .


Pantopon is prepared by treating standardized medicinal opium with  hydrochloric acid  or, more commonly, mixing 20 parts morphine HCl, 5 parts codeine, 6 parts thebaine, 8 parts noscapine, 2 parts narcotine, 6 parts miscellaneous alkaloids hydrochlorides. [ 4 ] [ 5 ]


Pantopon is, in other words,  opium  with all of the tar and other insolubles removed in an injectable form which, by weight, is nearly as potent as  morphine . It was invented in 1909 by the  Hoffmann-La Roche  pharmaceutical company. Other drugs of the same type have included  in the opium alkaloid  hydrobromides ,  sulfates ,  phosphates , and  valerates .  "Opium in a syringe " and "Injectable Whole Opium" were common advertising slogans for the product from Roche. An example of similar product to Pantopon is Omnopon, which contains morphine, codeine, and papaverine. [ 6 ]


Pantopon gave its name to the poem "Pantopon Rose" by the American writer  William Burroughs [ 7 ]  and to a song with the same name by the  Northern Ireland   alternative metal  band  Therapy?  on their 1994 album  Troublegum . Pantopon also gave its name to the 1996  Mexican  documentary  Rosa Pantopon . [ 8 ]


Papaveretum  ( BAN ) is a preparation containing a mixture of  hydrochloride  salts of  opium   alkaloids . [ 1 ]  Since 1993, papaveretum has been defined in the  British Pharmacopoeia  (BP) as a mixture of 253 parts  morphine hydrochloride , 23 parts  papaverine hydrochloride , and 20 parts  codeine hydrochloride . [ 2 ]  It is commonly marketed to medical agencies under the  trade name   Omnopon .


Although the use of papaveretum is now relatively uncommon following the wide availability of single-component  opiates  and synthetic  opioids  (e.g.  pethidine ), it is still used to relieve moderate to severe pain and for pre-operative sedation. In clinical settings, papaveretum is usually administered to patients via  subcutaneous ,  intramuscular , or  intravenous  routes. Additionally, the morphine  syrettes  found in combat medical kits issued to military personnel actually contain Omnopon.


Prior to 1993, papaveretum also contained  noscapine , though this component was removed from the BP formulation due to the  genotoxic  potential of noscapine.