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Arimoclomol
go.drugbank.com/drugs/DB05025Approved[L51389] It is also being investigated in amyotrophic lateral sclerosis (ALS) [A264414, A264429] and inclusion body myositis.[A264419] … [L51384] Arimoclomol was approved by the FDA on September 20, 2024, making it the first treatment for Niemann-Pick disease, type C (NPC).
Synonyms: 3-PYRIDINECARBOXIMIDOYL CHLORIDE, N-((2R)-2-HYDROXY-3-(1-PIPERIDINYL)PROPOXY), 1-OXIDE, N-[(2R)-2-Hydroxy-3-(1-piperidyl)propoxy]pyridine-3-carboximidoyl chloride, 1-oxideTriclabendazole
go.drugbank.com/drugs/DB12245ApprovedInvestigationalTriclabendazole was previously used in the treatment of fascioliasis in livestock, but is now approved for human use. … Triclabendazole, manufactured by Novartis pharmaceuticals, is an antihelminthic drug that was approved by the FDA in February 2019 for the treatment of fascioliasis in humans.
Apadamtase alfa
go.drugbank.com/drugs/DB15164ApprovedInvestigational[A262071] Apadamtase alfa, sold under the brand name Adzynma (ADAMTS13, recombinant-krhn), was approved by the FDA in November 2023 for use as an enzyme replacement therapy in patients with cTTP. … for cleaving large von Willebrand factor multimers and preventing the formation of microthrombi - this deficiency in ADAMTS13 is responsible for the microangiopathic complications associated with cTTP
Menadione
go.drugbank.com/drugs/DB00170ApprovedNutraceuticalA synthetic naphthoquinone without the isoprenoid side chain and biological activity, but can be converted to active vitamin K2, menaquinone, after alkylation in vivo.
Xanomeline
go.drugbank.com/drugs/DB15357ApprovedInvestigational[A264509] Advances in pre-clinical research and findings in clinical trials have led to a resurgence of interest in the cognition-enhancing potential of muscarinic agonists in schizophrenia, as it was … discovered that M1 and M4 muscarinic acetylcholine receptors are highly expressed in brain regions that are implicated in cognition.
Linerixibat
go.drugbank.com/drugs/DB11729ApprovedInvestigationalLinerixibat is a first-in-class and small-molecule inhibitor of the ileal bile acid transporter (IBAT). … [A275481, A275482] On March 19, 2026, linerixibat was approved by the FDA for the treatment of cholestatic pruritus in patients with primary biliary cholangitis (PBC).
Nesiritide
go.drugbank.com/drugs/DB04899ApprovedInvestigationalWithdrawnNesiritide is a 32 amino acid recombinant human B-type natriuretic peptide. … Nesiritide is a medication used to treat acutely decompensated congestive heart failure with dyspnea at rest or with minimal exertion (such as talk, eating or bathing).
Categories: Vasodilators Used in Cardiac DiseasesNickel sulfate
go.drugbank.com/drugs/DB14180ApprovedNickel sulfate is used in allergenic testing.
Categories: Compounds used in a research, industrial, or household settingSynonyms: nickel(II) sulfateMethylnaltrexone
go.drugbank.com/drugs/DB06800ApprovedInvestigationalIt is also a weak CYP2D6 inhibitor. FDA approved in 2008. … Methylnaltrexone is a pheriphally-acting μ-opioid antagonist that acts on the gastrointestinal tract to decrease opioid-induced constipation without producing analgesic effects or withdrawal symptoms.
Zavegepant
go.drugbank.com/drugs/DB15688ApprovedInvestigational[L45505] CGRP is released from sensory nerves and acts as a strong vasodilator, and thanks to these properties, it is involved in pain pathways. … [A258190,A258195] In March 2023, the FDA approved the use of zavegepant nasal spray for the acute treatment of migraine with or without aura in adults.
Synonyms: 1-piperidinecarboxamide, 4-(1,2-dihydro-2-oxo-3-quinolinyl)-n-((1r)-1-((7-methyl-1h-indazol-5-yl)methyl)-2-(4-(1-methyl-4-piperidinyl)-1-piperazinyl)-2-oxoethyl)-