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Treosulfan
go.drugbank.com/drugs/DB11678ApprovedInvestigational[L52245] It was approved by the EMA in June 2019[L16965] and by the FDA in January 2025 for use in combination with [fludarabine].[L52575] … Treosulfan is a prodrug alternative to [busulfan] for myeloablative conditioning prior to hematopoietic stem cell transplantation.
Remoxipride
go.drugbank.com/drugs/DB00409ApprovedWithdrawn[A215422] It gained approval in the UK in 1989 but was withdrawn in 1993 after it was found to be associated with an increased incidence of aplastic anemia.[A215422,A215512] … Remoxipride is an atypical antipsychotic agent that is specific for dopamine D<sub>2</sub> receptors.
Zenocutuzumab
go.drugbank.com/drugs/DB15559ApprovedInvestigational[L51983] In May 2026, zenocutuzumab was granted additional approval by the US FDA for use in NRG1-positive cholangiocarcinoma. … [A264833,A264843] Zenocutuzumab is also capable of promoting antibody-dependent cellular cytotoxicity in cancer cells.
Defactinib
go.drugbank.com/drugs/DB12282ApprovedInvestigational[L53208,A273943] Defactinib was approved by the US FDA in May 2025 in a co-package alongside [avutometinib] (called Avmapki Fakzynja) for the treatment of recurrent KRAS-mutated low-grade serous ovarian … resistance mechanism to MAPK inhibitors, facilitating tumor cell proliferation and survival in the absence of MAPK signaling.
Synonyms: N-methyl-4-[[4-[[3-[methyl(methylsulfonyl)amino]pyrazin-2-yl]methylamino]-5-(trifluoromethyl)pyrimidin-2-yl]amino]benzamideTideglusib
go.drugbank.com/drugs/DB12129ApprovedInvestigationalWithdrawnIt is reported to be a potent anti-inflammatory and neuroprotective that is a non-ATP competitive inhibitor of glycogen synthase kinase 3 (GSK-3). … [A31601] Tideglusib is being developed by the Spanish pharmaceutic company Zeltia group and its current status is withdrawn for the treatment of Alzheimer's disease as of 2012.
Pegzilarginase
go.drugbank.com/drugs/DB14780ApprovedFDA in February 2026 for the treatment of hyperargininemia in adult and pediatric patients aged 2 years and older with ARG1-D, to be used in conjunction with dietary protein restriction. … Pegzilarginase is a recombinant, cobalt-substituted, and pegylated human arginase 1 enzyme therapy designed to address the underlying metabolic deficit in arginase 1 deficiency (ARG1-D).
Synonyms: Optimised human arginase I, Co-ArgI-PEG modified human arginase IGinkgo biloba
go.drugbank.com/drugs/DB01381ApprovedInvestigationalNutraceutical[L32789] _Ginkgo biloba_ is an herbal plant that is now cultivated worldwide. It is originally native to China, and _ginkgo biloba_ extract has been used in traditional Chinese medicine for centuries. … _Ginkgo biloba_ is found in a number of homeopathic and over-the-counter herbal products and dietary supplements, but it has no approved therapeutic indications by regulatory bodies, such as the FDA, EMA
Norgestimate
go.drugbank.com/drugs/DB00957ApprovedInvestigationalNorgestimate was first described in the literature in 1977. … [A191068] It is commonly formulated with [ethinylestradiol] as a combined oral contraceptive that can also be used to treat moderate acne vulgaris.
Synonyms: (17α)-17-(Acetyloxy)-13-ethyl-18,19-dinorpregn-4-en-20-yn-3-one 3-oxime, (+)-13-Ethyl-17-hydroxy-18,19-dinor-17α-pregn-4-en-20-yn-3-one oxime acetate (ester)Vutrisiran
go.drugbank.com/drugs/DB16699ApprovedInvestigational[A249010] The Val30Met variant is the most prevalent among hereditary ATTR patients with polyneuropathy, especially in Portugal, France, Sweden, and Japan. … [L42085] Another siRNA indicated for the treatment of polyneuropathy associated with hereditary ATTR is [patisiran].[A249010] Vutrisiran was approved by the FDA in June 2022.
Selpercatinib
go.drugbank.com/drugs/DB15685ApprovedInvestigational[A202049, A202055, A202052, L13604] Although selpercatinib is currently still under investigation in clinical trial NCT04211337, it was granted accelerated FDA approval on May 8, 2020, for specific … Although multikinase inhibitors, including [cabozantinib], [ponatinib], [sorafenib], [sunitinib], and [vandetanib], have shown efficacy in RET-driven cancers, their lack of specificity is generally associated