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Pariglasgene brecaparvovec
go.drugbank.com/drugs/DB16801Approved[L64050] The FDA granted pariglasgene brecaparvovec-opnr accelerated approval on August 19, 2026, making it the first approved treatment for glycogen storage disease type Ia. … Pariglasgene brecaparvovec-opnr is an adeno-associated virus serotype 8 (AAV8)–based gene therapy for glycogen storage disease type Ia (GSDIa), a disorder caused by deficiency of the enzyme glucose-6-phosphatase
Suzetrigine
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]
Calcium levulinate
go.drugbank.com/drugs/DB13800ApprovedCalcium levulinate can be used alone, or with calcium lactate, calcium chloride, and other compounds, either for pharmaceutical tablets, capsules, or injections preparation [L2765].
Ethylenediamine
go.drugbank.com/drugs/DB14189ApprovedEthylenediamine is an organic compound that is used as a building block for the production of many other chemical products. … It is also used as an excipient in many pharmacological preparations such as creams. Notably, ethylenediamine is a contact sensitizer capable of producing local and generalized reactions [A34285].
Edrophonium
go.drugbank.com/drugs/DB01010ApprovedIt has also been used as an antidote to curare principles.
Ezetimibe
go.drugbank.com/drugs/DB00973ApprovedInvestigationalessential role in promoting intestinal cholesterol uptake via an ezetimibe-sensitive pathway. … [A15202] Ezetimibe is used as an adjunctive therapy to a healthy diet to lower cholesterol levels in primary hyperlipidemia, mixed hyperlipidemia, homozygous familial hypercholesterolemia (HoFH), and homozygous
Mixtures: EXOTIB-DUOProducts: Nra-ezetimibe, Nra-ezetimibe TabletsBetibeglogene autotemcel
go.drugbank.com/drugs/DB16900ApprovedInvestigationaldonor due to the risks of graft rejection, graft-versus-host disease, and other treatment-related toxic effects. … [A251770,A251785] Betibeglogene autotemcel was approved by the FDA in August 2022 and is the first ex-vivo lentiviral vector gene therapy available in the U.S. for the treatment of people with β-thalassemia
Aficamten
go.drugbank.com/drugs/DB18490ApprovedInvestigational[L54788] Approved by the FDA on December 19, 2025, aficamten is indicated for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and
Evolocumab
go.drugbank.com/drugs/DB09303ApprovedInvestigationalIt is a subcutaneous injection approved by the FDA for individuals on maximum statin therapy who still require additional LDL-cholesterol lowering. … It is approved for both homozygous and heterozygous familial cholesterolemia as an adjunct to other first-line therapies.
Chloroform
go.drugbank.com/drugs/DB11387ApprovedVet approvedWithdrawnChloroform is part of the FDA drug products withdrawn or removed from the market for reasons of safety or effectiveness.[L43942] … Chloroform is an organic small molecule, member of the family of the chloromethanes that presents a formula of CHCl3.