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Displaying drugs 376 - 400 of 1447 in total
Tofersen is under an intrathecally administered antisense oligonucleotide targeting the mutated SOD1 gene that causes amyotrophic lateral sclerosis (ALS). Although there were various causes of ALS, 2% of ALS cases are due to SOD1 mutations, with more than 200 SOD1 mutations documented.[A259028,A259033] Tofersen was granted accelerated approval from the FDA...
Approved
Investigational
Abrocitinib is an oral small-molecule inhibitor of Janus kinase 1 (JAK1). Janus kinases are intracellular enzymes involved in transduction pathways that regulate hematopoiesis and immune cell function. The Janus kinase (JAK)–signal transducer and activator of transcription (STAT) signalling pathway plays a central role in the pathogenesis of a variety of...
Approved
Investigational
Pemigatinib is a small molecule kinase inhibitor with antitumour activity. It works by inhibiting fibroblast growth factor receptors (FGFRs), which are receptor tyrosine kinases that activate signalling pathways in tumour cells. FGFRs gained attention as potential therapeutic targets in selected cancers, as FGFR gene alterations were observed in a wide...
Approved
Investigational
Enasidenib is an orally available treatment for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with specific mutations in the isocitrate dehydrogenase 2 (IDH2) gene, which is a recurrent mutation detected in 12-20% of adult patients with AML [A20344, A20345]. Patients eligible for this treatment...
Approved
Investigational
Coenzyme M (commonly known by its salt form, Mesna) is a synthetic sulfhydryl (thiol) compound and is used for prophylaxis of Ifosfamide and cyclophosphamide induced hemorrhagic cystitis.
Approved
Investigational
Omidenepag isopropyl is a topical ocular hypotensive agent used to reduce intraocular pressure (IOP) in patients with glaucoma and ocular hypertension. Omidenepag isopropyl is quickly metabolized to its active metabolite, omidenepag, a molecule with high selectivity and agonistic activity towards the prostaglandin E2 (EP2) receptor.[A253263,A253268] Prostanoid FP receptor agonists (FP...
Approved
Investigational
Retifanlimab is a humanized IgG4 kappa monoclonal antibody that binds to the programmed death receptor-1 (PD-1), blocking PD-1 interaction with its ligands, programmed death-ligand 1 (PD-L1) and programmed death-ligand 2 (PD-L2). By blocking the PD-1/PD-L1/2 pathway, retifanlimab potentiates T-cell activity and boosts the immune response against cancer cells. Other monoclonal...
Approved
Investigational
Approved
Matched Mixtures name: … Phoxillum B22k4/0
Approved
Investigational
Matched Iupac: … {1,26}.0^{3,8}.0^{23,27}.0^{25,42}.0^{32,44}.0^{39,43}.0^{37,45}]heptatetraconta-2(47),3,5,7,27,29(44 ... 2lambda5,9,19,26,43lambda5,44lambda5,45lambda5-heptaaza-15lambda5-phospha-1-cobaltadodecacyclo[27.14.1.1^{1,34}.1^{2,9}.1^{10,13}.0^ …
A 9,10alpha-dihydro derivative of ergotamine. Dihydroergotamine is used as an abortive therapy for migraines. Its use has largely been supplanted by triptans in current therapy due to the class's greater selectivity and more favourable side effect profile. Recent improvements have been made in the design of intranasal delivery devices allowing...
Approved
Investigational
Matched Iupac: … dioxo-3-oxa-6,9-diazatricyclo[7.3.0.0^{2,6}]dodecan-4-yl]-6-methyl-6,11-diazatetracyclo[7.6.1.0^{2,7}.0^ …
Fesoterodine is an antimuscarinic prodrug for the treatment of overactive bladder syndrome.
Approved
Lepirudin is a recombinant hirudin formed by 65 amino acids that acts as a highly specific and direct thrombin inhibitor.[L41539,L41569] Natural hirudin is an endogenous anticoagulant found in Hirudo medicinalis leeches. Lepirudin is produced in yeast cells and is identical to natural hirudin except for the absence of sulfate on...
Approved
Withdrawn
Buprenorphine is a weak partial mu-opioid receptor agonist and a weak kappa-opioid receptor antagonist used for the treatment of severe pain.[A186283,A186292] It is also commonly used as an alternative to methadone for the treatment of severe opioid addiction. Buprenorphine is commercially available as the brand name product Suboxone which is...
Approved
Illicit
Investigational
Vet approved
Matched Iupac: … {1,6}.0^{6,14}.0^{7,12}]icosa-7,9,11-trien-11-ol ... cyclopropylmethyl)-16-[(2S)-2-hydroxy-3,3-dimethylbutan-2-yl]-15-methoxy-13-oxa-3-azahexacyclo[13.2.2.1^{2,8}.0^ …
Vutrisiran is a double-stranded small interfering ribonucleic acid (siRNA) that targets wild-type and mutant transthyretin (TTR) messenger RNA (mRNA). This siRNA therapeutic is indicated for the treatment of neuropathies associated with hereditary transthyretin-mediated amyloidosis (ATTR), a condition caused by mutations in the TTR gene. More than 130 TTR mutations have...
Approved
Investigational
Ceftobiprole medocaril is a prodrug of ceftobiprole, a fifth-generation semisynthetic cephalosporin antibacterial. Ceftobiprole is a broad-spectrum agent with demonstrated activity against both Gram-positive and Gram-negative bacteria, including antibiotic-resistant strains of Staphylcoccus aureus (methicillin-resistant Staphylococcus aureus; MRSA). The EMA's Committee for Medicinal Products for Human Use (CHMP) adopted a negative opinion...
Approved
Experimental
Investigational
Ranibizumab is a recombinant humanized IgG1 kappa isotype monoclonal antibody fragment directed against human vascular endothelial growth factor A (VEGF-A), which is a glycoprotein implicated in the pathophysiology of age-related macular degeneration.[A2301, L38978] Ranibizumab is used to treat various ocular disorders with abnormal growth of blood vessels, such as neovascular...
Approved
Semaglutide is a glucagon-like peptide 1 (GLP-1) analog used to manage type 2 diabetes along with lifestyle changes, such as dietary restrictions and increased physical activity.[A31421,L8681] Other members of this drug class include Exenatide and Liraglutide. Semaglutide was developed by Novo Nordisk and approved by the FDA for subcutaneous injection...
Approved
Investigational
Nalmefene, a 6-methylene analogue of naltrexone, is an opioid receptor antagonist. It acts as an antagonist at the mu (μ)-opioid and delta (δ)-opioid receptors and a partial agonist at the kappa (κ)-opioid receptor. In Europe, nalmefene oral tablets are used to reduce alcohol consumption in adults with alcohol dependence. Nalmefene...
Approved
Investigational
Withdrawn
Matched Iupac: … (1S,5R,13S,17S)-4-(cyclopropylmethyl)-14-methylidene-12-oxa-4-azapentacyclo[9.6.1.0^{1,13}.0^{5,17}.0
Matched Salts cas: … 58895-64-0
An opioid analgesic with actions and uses similar to those of morphine, apart from an absence of cough suppressant activity. It is used in the treatment of moderate to severe pain, including pain in obstetrics. It may also be used as an adjunct to anesthesia (From Martindale, The Extra Pharmacopoeia,...
Approved
Investigational
Vet approved
Matched Iupac: … (1S,5R,13R,17S)-10,17-dihydroxy-4-methyl-12-oxa-4-azapentacyclo[9.6.1.0^{1,13}.0^{5,17}.0^{7,18}]octadeca …
Risankizumab is a fully humanized IgG1 monoclonal antibody (mAb) directed against interleukin 23 (IL-23). It gained its first global approval in Japan in March 2019, followed by approval in Canada, the US, and Europe in April 2019. Risankizumab is used to treat plaque psoriasis, psoriatic arthritis, and Crohn's disease.[L39885,L44191,L44231] Risankizumab...
Approved
Investigational
Tebentafusp is a gp100 peptide-HLA-directed CD3 T cell engager. It is a bispecific, fusion protein and first-in-class drug of immune-mobilizing monoclonal T cell receptors against cancer (ImmTACs), a recently developed cancer immunotherapy with a novel mechanism of action. ImmTACs bind to target cancer cells that express a specific antigen of...
Approved
Investigational
The relief of pain (analgesia) is a primary goal for enhancing the quality of life of patients and for increasing the ability of patients to engage in day to day activities. Codeine, an opioid analgesic, was originally approved in the US in 1950 and is a drug used to decrease...
Approved
Illicit
Matched Iupac: … (1S,5R,13R,14S,17R)-10-methoxy-4-methyl-12-oxa-4-azapentacyclo[9.6.1.0^{1,13}.0^{5,17}.0^{7,18}]octadeca …
Mavorixafor is a CXC chemokine receptor 4 (CXCR4) antagonist. It was first approved by the FDA on April 30, 2024, for the treatment of warts, hypogammaglobulinemia, infections, and myelokathexis (WHIM) syndrome, a genetic immunodeficiency disorder characterized by a reduced number of mature neutrophils and lymphocytes. WHIM syndrome is caused by...
Approved
Investigational
Metamizole (dipyrone) is a pyrazolone derivative that belongs to the group of nonacid nonopioids. It is considered a potent analgesic and antipyretic with favourable gastrointestinal tolerability. Metamizole was formerly marketed in the US as Dimethone tablets and injection, Protemp oral liquid, and other drug products, and was withdrawn due to...
Approved
Investigational
Withdrawn
Matched Salts cas: … 68-89-3 ... 5907-38-0
CD20, an antigen expressed by various B and T cells, is an attractive therapeutic target in various cancers and autoimmune conditions.[A244195, A244200, A244205] Monoclonal antibodies for B cell depletion rely on their binding affinity and ability to clear bound cells through mechanisms such as antibody-dependent cellular cytotoxicity (ADCC), mediated by...
Approved
Investigational
Matched Description: … [A244210] Ublituximab is a chimeric anti-CD20 IgG1κ antibody produced in the rat YB2/0 cell line to target …
Displaying drugs 376 - 400 of 1447 in total