FDA Approves First Gene Therapy for Adults with Severe Hemophilia A
The U.S. Food and Drug Administration approved Roctavian, an adeno-associated virus vector-based gene therapy for the treatment of adults with severe hemophilia A without pre-existing antibodies to adeno-associated virus serotype 5 detected by an FDA-approved test.
Hemophilia A is a rare genetic bleeding disorder that occurs due to a mutation on the gene which produces factor VIII (FVIII), a protein that enables blood to clot.
Roctavian is a one-time gene therapy product administered as a single dose by intravenous infusion. Roctavian consists of a viral vector carrying a gene for clotting Factor VIII. The gene is expressed in the liver to increase blood levels of FVIII and reduce the risk of uncontrolled bleeding.
The safety and effectiveness of Roctavian were evaluated in a multinational study in adult men 18 to 70 years of age with severe Hemophilia A who were previously treated with Factor VIII replacement therapy.
Effectiveness was established based on results from a cohort of 112 patients followed up for at least 3 years after Roctavian treatment. Following the infusion, the mean annualized bleeding rate decreased from 5.4 bleeds per year at baseline to 2.6 bleeds per year.
The most common adverse reactions associated with Roctavian included mild changes in liver function, headache, nausea, vomiting, fatigue, abdominal pain and infusion-related reactions.
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