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Elexacaftor
go.drugbank.com/drugs/DB15444ApprovedInvestigational[A187361] The triple combination product Trikafta<sup>TM</sup>, manufactured by Vertex Pharmaceuticals, is the first product approved for the treatment of CF in individuals who are either homo- _or_ heterozygous … [A187361] It received FDA approval in October 2019 in combination with [tezacaftor] and [ivacaftor] as the combination product Trikafta<sup>TM</sup>.
Tazemetostat
go.drugbank.com/drugs/DB12887ApprovedInvestigational[L11476] Tazemetostat was first named in literature as EPZ-6438.[A190363] Tazemetaostat was granted FDA approval on 23 January 2020.[L11476]
Bremelanotide
go.drugbank.com/drugs/DB11653Approved[A179683] Since then it was investigated for its place in treating sexual dysfunction in men and women but is now only indicated for women. … [L9635] Bremelanotide was first described in the literature in 2003 when it was known by the investigational code PT-141.
Busulfan
go.drugbank.com/drugs/DB01008ApprovedInvestigationalAccording to the Fourth Annual Report on Carcinogens (NTP 85-002, 1985), busulfan is listed as a known carcinogen. … Busulfan is a bifunctional alkylating agent, having a selective immunosuppressive effect on bone marrow. It is not a structural analog of the nitrogen mustards.
Dabrafenib
go.drugbank.com/drugs/DB08912ApprovedInvestigational[L41955] In May 2018, Tafinlar (dabrafenib), in combination with Mekinist ([DB08911]), was approved to treat anaplastic thyroid cancer caused by an abnormal BRAF V600E gene.[L41955] … It was approved on May 29, 2013, for the treatment of melanoma with V600E or V6000K mutation.[L41955] It was also used for metastatic non-small cell lung cancer with the same mutation.
Mafenide
go.drugbank.com/drugs/DB06795ApprovedVet approvedWithdrawnIn November 2022, the use of mafenide acetate (powder for 5% topical solution) was withdrawn by the FDA due to an unresolved confirmatory study required to fulfill the accelerated approval requirements … [L36773] In 1998, mafenide acetate was approved under the FDA’s accelerated approval regulations.
Penicillamine
go.drugbank.com/drugs/DB00859ApprovedInvestigationalIt is an α-amino acid metabolite of penicillin, although it has no antibiotic properties.
Categories: Compounds used in a research, industrial, or household settingSuzetrigine
go.drugbank.com/drugs/DB18927ApprovedInvestigational[A264953] On January 30, 2025, suzetrigine was approved by the FDA as a first-in-class non-opioid analgesic.[L52098] … Suzetrigine is a Na<sub>V</sub>1.8 voltage-gated sodium channel blocker with analgesic properties[A264948] Na<sub>V</sub>1.8 is abundantly expressed in peripheral sensory nerves to play a role in pain
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
Nerandomilast
go.drugbank.com/drugs/DB18237ApprovedInvestigational[L54126] On October 7, 2025, nerandomilast was approved as the first new treatment for idiopathic pulmonary fibrosis in more than a decade. … [A274581] On December 19, 2025, the U.S. FDA approved nerandomilast tablets for the treatment of progressive pulmonary fibrosis (PPF) in adults.