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Atidarsagene autotemcel
go.drugbank.com/drugs/DB17538ApprovedInvestigational[L45191] Libmeldy was granted orphan designation by the EMA in April 2007, and was issued a marketing authorization in the EU in December 2020 for the treatment of certain manifestations of metachromatic … [L45196] It was granted FDA approval under the brand name Lenmeldy in March 2024.[L50256]
Synonyms: Autologous CD34+ cell enriched population that contains haematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A (ARSA) geneViomycin
go.drugbank.com/drugs/DB06827ApprovedThese drugs bind RNA in bacterial ribosomes and inhibit protein synthesis. Viomycin was derived from the actinomycete _Streptomyces puniceus_.
Bismuth subnitrate
go.drugbank.com/drugs/DB13209ApprovedWithdrawnThe use of bismuth substrate as an active ingredient in over-the-counter antacids is approved by the FDA.
Olutasidenib
go.drugbank.com/drugs/DB16267ApprovedInvestigationalin 2-hydroxyglutarate (2-HG), a metabolite that participates in tumerogenesis. … [L44256,L44261] It is indicated for the treatment of relapsed or refractory acute myeloid leukemia (AML) in patients with a susceptible IDH1 mutation as determined by an FDA-approved test.
Alemtuzumab
go.drugbank.com/drugs/DB00087ApprovedInvestigationalAlemtuzumab is produced in mammalian cell (Chinese hamster ovary) suspension culture in a medium containing neomycin.[L43397] Alemtuzumab was approved by the FDA in 2001. … The Campath-1H antibody is an IgG1 kappa with the human variable framework and constant regions, and complementarity-determining regions from a murine (rat) monoclonal antibody (Campath-1G).
Palovarotene
go.drugbank.com/drugs/DB05467ApprovedFibrodysplasia Ossificans Progressiva (FOP), with an estimated worldwide prevalence of one in 2 million individuals, is an exceptionally rare genetic disorder. … [A244920] Palovarotene was approved for use in Canada in January 2022 for the management of heterotopic ossification (HO) in patients with FOP, representing the first global approval for any FOP therapy
Inavolisib
go.drugbank.com/drugs/DB15275ApprovedInvestigationalInavolisib is an inhibitor of PI3Kα that was approved by the FDA in October 2024 for the treatment of certain patients with advanced breast cancers. … [L51748,L51738] In February 2025, it was approved by Health Canada for the same indication. [L52605]
Ceritinib
go.drugbank.com/drugs/DB09063ApprovedInvestigationalFollowing treatment with crizotinib (a first-generation ALK inhibitor), most tumours develop drug resistance due to mutations in key "gatekeeper" residues of the enzyme. … The FDA approved ceritinib in April 2014 due to a surprisingly high response rate (56%) towards crizotinib-resistant tumours and has designated it with orphan drug status.
Synonyms: N-{2-[(5-chloro-2-{[2-isopropoxy-5-methyl-4-(piperidin-4-yl)phenyl]amino}pyrimidin-4-yl)amino]phenyl}propane-2-sulfonamidePipradrol
go.drugbank.com/drugs/DB11584ApprovedWithdrawnIn addition to this, it has shown favorable effects in postpartum depressive symptoms [L2516]. … The social and in particular acute clinical harms of pipradrol derivatives have led to their control under the Misuse of Drugs Act 1971 in the UK in 2012 [T178].
Rezafungin
go.drugbank.com/drugs/DB16310ApprovedInvestigational[L45633,L45658] It was subsequently approved in the EU in November of the same year. [L52745] … Rezafungin is an echinocandin antifungal drug.