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Vimseltinib
go.drugbank.com/drugs/DB17520ApprovedInvestigational[L52230,A265035] Vimseltinib - under the brand name Romvimza - was approved by the FDA in February 2025 for the treatment of tenosynovial giant cell tumours for which surgical resection is not appropriate
Nifurtimox
go.drugbank.com/drugs/DB11820ApprovedInvestigational[L15366] Nifurtimox, developed by Bayer, is a nitrofuran antiprotozoal drug used in the treatment of Chagas disease. … Chagas disease, caused by a parasite known as Trypanosoma cruzi (T.cruzi), is a vector-transmitted disease affecting animals and humans in the Americas.
Temsirolimus
go.drugbank.com/drugs/DB06287ApprovedInvestigationalIt was developed by Wyeth Pharmaceuticals under the trade name Torisel. Temsirolimus was approved by the FDA in late May 2007 as well as the European Medicines Agency (EMEA) on November 2007.
Narsoplimab
go.drugbank.com/drugs/DB16418ApprovedInvestigational[A241045, A241050] Approved by the FDA on December 24, 2025, narsoplimab-wuug is indicated for the treatment of hematopoietic stem cell transplant–associated thrombotic microangiopathy (TA-TMA) in adults
Glucarpidase
go.drugbank.com/drugs/DB08898ApprovedInvestigational[L39855] Glucarpidase was first approved by the FDA in January 2012,[A7476] followed by the European Commission's approval in January 2022.[L39865] It is marketed as VORAXAZE. … Glucarpidase is a recombinant carboxypeptidase G2 produced by genetically modified _Escherichia coli_ bacteria. It is a 390-amino acid homodimer protein.
Fostamatinib
go.drugbank.com/drugs/DB12010ApprovedInvestigationalFostamatinib has also been granted orphan drug status by the FDA [L2644].
Cannabidivarin
go.drugbank.com/drugs/DB14050InvestigationalCompared to its homolog, [DB09061], CBDV is shortened by two methyl (CH2) groups on its side chain. … In October 2017 CBDV was given orphan designation by the European Medicines Agency for use in Rett Syndrome [L2952] and again in February 2018 for treatment of Fragile X Syndrome [L2951].
Corifollitropin alfa
go.drugbank.com/drugs/DB09066ApprovedInvestigationalCorifollitropin alfa is produced by a method known as ‘recombinant DNA technology’. … This means that it is made by cells into which a DNA has been introduced that makes them able to produce corifollitropin alfa [L2270].
Etuvetidigene Autotemcel
go.drugbank.com/drugs/DB17593ApprovedInvestigational[L54693] WAS is a rare X-linked disorder caused by a mutation in the gene encoding the WAS protein, leading to a compromised immune system. … [L54698] First approved by the FDA on December 9, 2025, etuvetidigene autotemcel is used to treat Wiskott–Aldrich syndrome (WAS) in selected pediatric and adult patients.
Antihemophilic factor, human recombinant
go.drugbank.com/drugs/DB00025ApprovedInvestigationalHuman recombinant antihemophilic factor (AHF) or Factor VIII, 2332 residues, glycosylated, produced by CHO cells