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Sepiapterin
go.drugbank.com/drugs/DB16326ApprovedInvestigational[L53548,L53593] Sepiapterin was granted marketing authorization by the European Commission in June 2025 and by the US FDA in July 2025 for the treatment of adults and children with phenylketonuria. … Sepiapterin is a small molecule activator of phenylalanine hydroxylase (PAH) used to reduce phenyalanine levels in patients with phenylketonuria.
Betibeglogene autotemcel
go.drugbank.com/drugs/DB16900ApprovedInvestigational[A251770,A251785] Betibeglogene autotemcel was approved by the FDA in August 2022 and is the first ex-vivo lentiviral vector gene therapy available in the U.S. for the treatment of people with β-thalassemia … [L42940] β-thalassemia is a condition caused by mutations in the β-globin gene (HBB) that leads to a significant decrease in the production of β-globin.
Vorapaxar
go.drugbank.com/drugs/DB09030ApprovedBy inhibiting PAR-1, a thrombin receptor expressed on platelets, vorapaxar prevents thrombin-related platelet aggregation. … Vorapaxar is a tricyclic himbacine-derived selective inhibitor of protease activated receptor (PAR-1) indicated for reducing the incidence of thrombotic cardiovascular events in patients with a history
Synonyms: Carbamic acid, N-[(1R,3aR,4aR,6R,8aR,9S,9aS)-9-[(E)-2-[5-(3-fluorophenyl)-2-pyridinyl]ethenyl]dodecahydro-1-methyl-3-oxonaphtho[2,3-c]furan-6-yl]-, ethyl ester, Ethyl N-[(3R,3aS,4S,4aR,7R,8aR,9aR)-4-[(E)-2-[5-(3-fluorophenyl)-2-pyridyl]vinyl]-3-methyl-1-oxo-3a,4,4a,5,6,7,8,8a,9,9a-decahydro-3H-benzo[f]isobenzofuran-7-yl]carbamateHistrelin
go.drugbank.com/drugs/DB06788ApprovedInvestigationalWithdrawnAs the product Supprelin LA, histrelin is indicated for the treatment of CPP in children (approved by the FDA in May 2007).[L41700] … [L41715] Vantas was later discontinued by Endo Pharmaceuticals Inc. on September 21, 2021.
Synonyms: 5-oxo-L-prolyl-L-histidyl-L-tryptophyl-L-seryl-L-tyrosyl-1-benzyl-D-histidyl-L-leucyl-L-arginyl-N-ethyl-L-prolinamide, 5-oxo-L-prolyl-L-histidyl-L-tryptophyl-L-seryl-L-tyrosyl-Nτ-benzyl-D-histidyl-L-leucyl-L-arginyl-N-ethyl-L-prolinamideIsoflurophate
go.drugbank.com/drugs/DB00677ApprovedWithdrawnIts vapor is highly toxic and it is recommended that only solutions in arachis oil be used therapeutically. (From Martindale, The Extra Pharmacopoeia, 29th ed, p1330) … An irreversible cholinesterase inhibitor with actions similar to those of echothiophate. It is a powerful miotic used mainly in the treatment of glaucoma.
Prademagene zamikeracel
go.drugbank.com/drugs/DB17895ApprovedInvestigational[A273833,A273838] Patient cells are then transduced ex vivo with a replication-incompetent retroviral vector (RVV) containing the full-length COL7A1 gene, resulting in cells that express functional collagen … [L53013] It was granted an Orphan Drug designation by the EMA.[A273833]
Synonyms: EB 101, EB-101Labetalol
go.drugbank.com/drugs/DB00598ApprovedInvestigational[L7730] Labetalol is formulated as an injection or tablets to treat hypertension.[L7727,L7730] Labetalol was granted FDA approval on 1 August 1984.[L7724]
Products: Labetalol HCl in Dextrose, Labetalol HCl in Sodium ChlorideLanreotide
go.drugbank.com/drugs/DB06791ApprovedInvestigationalIt is approved in several countries worldwide, including the United Kingdom, Australia, and Canada. Lanreotide was first approved for use in the United States by the FDA on August 30, 2007. … Lanreotide is a drug employed in the management of acromegaly (a hormonal condition caused by excess growth hormone) in addition to symptoms caused by neuroendocrine tumors, especially carcinoid syndrome
Products: Mytolac 60 mg Injektionslösung in einer Fertigspritze, Mytolac 90 mg Injektionslösung in einer FertigspritzeLevocarnitine
go.drugbank.com/drugs/DB00583ApprovedInvestigationalIt is used therapeutically to stimulate gastric and pancreatic secretions and in the treatment of hyperlipoproteinemias.
Synonyms: 3-Carboxy-2-hydroxy-N,N,N-trimethyl-1-propanaminium hydroxide, inner saltTafamidis
go.drugbank.com/drugs/DB11644ApprovedInvestigational[A189717] Tafamidis was granted an EMA market authorisation on 16 November 2011[L6247] and FDA approval on 3 May 2019.[L11280]