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Obeticholic acid
go.drugbank.com/drugs/DB05990ApprovedInvestigational[A18696] In 2016, it was granted approval to treat primary biliary cholangitis in combination with [ursodeoxycholic acid], which was previously the mainstay treatment for this condition. … The NDA from Intercept Pharmaceuticals was approved in November 2019 and obeticholic acid is expected to be granted full approval for this indication in 2020.[L12636]
Macimorelin
go.drugbank.com/drugs/DB13074ApprovedThey include treatment for growth retardation in children and cachexia associated with chronic disease such as AIDS and cancer. … Macimorelin, developed by Aeterna Zentaris, was approved by the FDA in December 2017 under the market name Macrilen for oral solution.
Mavorixafor
go.drugbank.com/drugs/DB05501ApprovedInvestigational[L50642] It was first approved by the FDA on April 30, 2024, for the treatment of warts, hypogammaglobulinemia, infections, and myelokathexis (WHIM) syndrome, a genetic immunodeficiency disorder characterized … [L50642] As CXCR4 mutations have also been implicated in human immunodeficiency virus (HIV), Waldenstrom’s macroglobulinemia (WM), B-cell non-Hodgkin lymphoma, and solid tumours, including melanoma,
Suvorexant
go.drugbank.com/drugs/DB09034ApprovedInvestigationalIt has been approved for the treatment of insomnia.
Anti-inhibitor coagulant complex
go.drugbank.com/drugs/DB13151ApprovedInvestigationalAnti-inhibitor coagulant complex, also known as FEIBA (factor eight inhibitor bypassing activity), contains several proteins involved in the prothrombinase complex.
Dorocubicel
go.drugbank.com/drugs/DB19448Approved[L54106] Dorocubicel, as a component of Zemcelpro, was granted conditional marketing authorization in the European Union in August 2025 for use in allogeneic hematopoietic stem cell transplantation
mRNA-1283
go.drugbank.com/drugs/DB22668ApprovedInvestigational[L53408] As opposed to previously approved COVID-19 mRNA vaccines, including the original [Moderna COVID-19 Vaccine], mRNA-1283 encodes only the immunodominant regions (NTD and RBD) of the SARS-CoV- … [L53413,A274083] mNEXSPIKE was approved by the US FDA in June 2025 for use in patients who have been previously vaccinated with any COVID-19 vaccine and who are at a higher risk of severe outcomes from
Fosdenopterin
go.drugbank.com/drugs/DB16628Approved[A230088,A230593] In the past, management strategies for this disorder involved symptomatic and supportive treatment,[L32163] though efforts were made to develop a suitable exogenous replacement for the … In 2009 a recombinant, E. coli-produced cPMP was granted orphan drug designation by the FDA, becoming the first therapeutic option for patients with MoCD type A.
Avapritinib
go.drugbank.com/drugs/DB15233ApprovedInvestigationalAvapritinib was granted FDA approval on 9 January 2020 [L40363] and EMA approval on 24 September 2020.[L41464]
Aspartame
go.drugbank.com/drugs/DB00168InvestigationalNutraceuticalFlavoring agent sweeter than sugar, metabolized as phenylalanine and aspartic acid.