Roctavian FDA Approval History
Last updated by Judith Stewart, BPharm on July 3, 2023.
FDA Approved: Yes (First approved June 29, 2023)
Brand name: Roctavian
Generic name: valoctocogene roxaparvovec-rvox
Dosage form: Suspension for Intravenous Infusion
Company: BioMarin Pharmaceutical Inc.
Treatment for: Hemophilia A
Roctavian (valoctocogene-roxaparvovec-rvox) is an adeno-associated virus vector-based gene therapy for the treatment of adults with severe hemophilia A.
- Roctavian is indicated for the treatment of adults with severe hemophilia A (congenital factor VIII deficiency with factor VIII activity < 1 IU/dL) without pre-existing antibodies to adeno-associated virus serotype 5 detected by an FDA-approved test.
- Hemophilia A is a lifelong condition caused by a mutation in the F8 gene which responsible for producing a protein called FVIII, one of the essential blood proteins necessary for blood clotting. When severely deficient in FVIII, people with hemophilia A are at risk for painful and potentially life-threatening bleeds, which can occur spontaneously. The current standard of care for hemophilia A is preventative therapy that involves receiving infusions or injections to maintain enough clotting factor in the bloodstream to prevent bleeds.
- Roctavian is a gene therapy that is designed to replace the function of the mutated F8 gene, allowing people with severe hemophilia A to produce their own FVIII and thereby limit bleeding episodes.
- Roctavian is administered as a single-dose intravenous infusion.
- Warnings and precautions associated with Roctavian include infusion-related reactions, hepatotoxicity, thromboembolic events, and hepatocellular malignancy.
- Common adverse reactions include nausea, fatigue, headache, infusion-related reactions, vomiting, and abdominal pain.
Common laboratory abnormalities include ALT, aspartate aminotransferase (AST), lactate dehydrogenase (LDH), creatine phosphokinase (CPK), factor VIII activity levels, gamma-glutamyl transferase (GGT) and bilirubin above normal limits.
Development timeline for Roctavian
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