Roctavian is a prescription medication approved by the U.S. Food and Drug Administration (FDA) to treat adults with severe hemophilia A. Roctavian is also known by its drug name, valoctocogene roxaparvovec-rvox.
Roctavian is a gene-replacement therapy believed to work by providing the gene for clotting factor VIII that’s missing in people with hemophilia A. Roctavian is delivered via an adeno-associated virus vector. To be eligible for treatment with Roctavian, you must first be tested for antibodies (immune proteins) against adeno-associated virus serotype 5 using an FDA-approved test. You must test negative for these antibodies to be eligible for Roctavian treatment.
How do I take it?
Roctavian is given as a one-time intravenous infusion.
The FDA-approved label for Roctavian lists common side effects including nausea, fatigue, headache, infusion reactions, vomiting, and abdominal pain.
Rare but serious side effects listed for Roctavian include severe infusion reactions, liver damage, blood clots, and the potential for developing liver cancer in people with preexisting risk factors.
For more details about this treatment, visit:
Roctavian — BioMarin
FDA Approves First Gene Therapy for Adults With Severe Hemophilia A — U.S. Food and Drug Administration (FDA)