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Ofatumumab
go.drugbank.com/drugs/DB06650ApprovedInvestigational[A6921] Ofatumumab was first approved by the FDA in 2009. … [L12753] The FDA subsequently approved ofatumumab for the treatment of RMS on August 20, 2020.
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. … [A262076] It may be inherited (congenital TTP; cTTP) or acquired due to autoantibodies (immune-mediated TTP; iTTP), although cTTP accounts for <5% of all cases of TTP.
Imetelstat
go.drugbank.com/drugs/DB14909ApprovedInvestigational[A187838] Imetelstat received FDA approval in June 2024 for use in certain patients with MDS who have transfusion-dependent anemia and are unable to use ESAs.[L50883,L50888] … [erythropoietin]), but primary resistance to these agents is frequent - the median response duration is 18 to 24 months, with most responders (70%) relapsing due to loss of sensitivity of erythroid progenitors
Oliceridine
go.drugbank.com/drugs/DB14881ApprovedInvestigational[A218026, A218031, A218051, A218056, A218061, A218066, A218071, L15516] Oliceridine was first reported in 2013,[A218026, A218086] but was initially not approved by the FDA due to concerns raised by … [A218041] Oliceridine gained FDA approval on August 7, 2020, and is currently marketed by Trevena Inc as OLINVYK™.[L15516]
Metreleptin
go.drugbank.com/drugs/DB09046ApprovedInvestigational[L41315] In February 2014, metreleptin was approved by the FDA for the treatment of complications of leptin deficiency, as an adjunct to diet, in patients with congenital generalized or acquired generalized … Metreleptin, a recombinant analog of the human hormone leptin, is an orphan drug used to treat complications of leptin deficiency in people with lipodystrophy.
Temozolomide
go.drugbank.com/drugs/DB00853ApprovedInvestigational[L32033] Temozolomide was granted FDA approval on August 11, 1999, as an oral capsule and subsequently on February 27, 2009, as an intravenous injection. … [A229853, A229923] Temozomolide as an adjunct to radiotherapy followed by maintenance dosing remains the standard of care for both Glioblastoma and refractory anaplastic astrocytoma.
Synonyms: 8-carbamoyl-3-methylimidazo(5,1-d)-1,2,3,5-tetrazin-4(3H)-one, 3,4-dihydro-3-methyl-4-oxoimidazo(5,1-d)-as-tetrazine-8-carboxamidePentoxyverine
go.drugbank.com/drugs/DB11186ApprovedWithdrawnPentoxyverine acts on sigma-1 receptors, as well as kappa and mu-opioid receptors. The FDA withdrew the use of all oral gel drug products containing pentoxyverine citrate. … It is an active ingredient in over-the-counter cough suppressants in combination with guaifenesin and H1-receptor antagonists [A32160].
Mixtures: Exall D, Zotex-DPralnacasan
go.drugbank.com/drugs/DB04875ExperimentalPralnacasan is an orally bioavailable pro-drug of a potent, non-peptide inhibitor of interleukin-1beta converting enzyme (ICE).
Formestane
go.drugbank.com/drugs/DB08905ApprovedWithdrawnIt is not US FDA approved, and the intramuscular injection form of formestane (Lentaron) which was approved in Europe has been withdrawn. … Formestane is also a prohormone of 4-hydroxytestosterone, an active steroid with weak androgenic activity and mild aromatase inhibitor activity.
Lonafarnib
go.drugbank.com/drugs/DB06448ApprovedInvestigational[L23414, L23544] Lonafarnib was granted FDA approval on November 20, 2020, and is the first FDA-approved treatment for HGPS and other related progeroid laminopathies.[L23414, L23549] … [A224379, A224384, A224389, A224394, A224399] Mechanistically, HGPS is underpinned by a single heterozygous C-to-T mutation at position 1824 of the _LMNA_ gene, which results in the accumulation of an