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Mitapivat
go.drugbank.com/drugs/DB16236ApprovedInvestigational[A245478] On January 5, 2026, the FDA also approved the use of in the treatment of anemia in adults with alpha- or beta-thalassemia, making it the first oral treatment for this condition.[L54953] … [A245478] On February 17, 2022, the FDA approved mitapivat as the first disease-modifying treatment for hemolytic anemia in adults with pyruvate kinase (PK) deficiency, a rare, inherited disorder leading
Oveporexton
go.drugbank.com/drugs/DB21680Approved[L64069] The FDA approved oveporexton on August 5, 2026 for the treatment of narcolepsy type 1 (narcolepsy with cataplexy) in adults. … [L64068,L64069] It is the first orexin receptor agonist approved by the FDA and the first medicine to treat NT1 by targeting its underlying cause rather than individual symptoms.
Ensifentrine
go.drugbank.com/drugs/DB16157ApprovedInvestigational[L50903] On June 26, 2024, the FDA announced the approval of an inhaled product of ensifentrine for the treatment of chronic obstructive pulmonary disease (COPD) in adults, which allows the drug to be
Vemurafenib
go.drugbank.com/drugs/DB08881ApprovedInvestigational[A31270] Vemurafenib was co-developed by Roche and Plexxikon and it obtained its FDA approval on August 17, 2011, under the company Hoffmann La Roche.
Deutivacaftor
go.drugbank.com/drugs/DB15141ApprovedInvestigational[L52275,A179677] Deutivacaftor was first approved by the US FDA in December 2024 in combination with two CFTR correctors - [tezacaftor] and [vanzacaftor] - for the treatment of patients with responsive
Remibrutinib
go.drugbank.com/drugs/DB16852ApprovedInvestigationalThe FDA approved remibrutinib on September 30, 2025 as the only oral, targeted treatment for chronic spontaneous urticaria.
Glofitamab
go.drugbank.com/drugs/DB16371ApprovedInvestigationalIn June 2023, the FDA approved the use of glofitamab for the treatment of patients with relapsed or refractory DLBCL not otherwise specified or large B-cell lymphoma (LBCL) arising from follicular lymphoma
MUC1 Dendritic Cell Vaccine
go.drugbank.com/drugs/DB05477InvestigationalDendritic Cell (DC) Therapy is a newly emerging and potent form of immune therapy that amplifies the actions of the body's own immune system to help fight cancer, AIDS and other serious conditions.
Eldecalcitol
go.drugbank.com/drugs/DB05295InvestigationalEldecalcitol (ED-71), a vitamin D analog, is a more potent inhibitor of bone resorption than alfacalcidol in an estrogen-deficient rat model of osteoporosis.
Elacytarabine
go.drugbank.com/drugs/DB05494InvestigationalElacytarabine is a fatty acid derivative of cytarabine, an approved cytotoxic cancer drug. Cytarabine has limitations such as minimal uptake in solid tumours and is only used to treat leukaemia. Elacytarabine is designed to overcome this...