FDA approves gene therapy for adults with severe hemophilia A

June 30, 2023
The FDA granted approval of Roctavian to BioMarin Pharmaceutical Inc.

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.

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 majority of patients who received Roctavian received corticosteroids to suppress the immune system for the gene therapy to be effective and safe. Treatment response to Roctavian may decrease over time. 

The most common adverse reactions associated with Roctavian included mild changes in liver function, headache, nausea, vomiting, fatigue, abdominal pain and infusion-related reactions. Close monitoring for infusion-related reactions and liver enzyme elevation is advised with Roctavian administration. In some cases, treatment with Roctavian was observed to increase FVIII activity levels above the normal limits. An increase in FVIII activity may increase the risk of thromboembolic events (blood clots that can cause harm by blocking blood flow). Introduction of the Roctavian product’s DNA sequence may carry the theoretical risk of developing hepatocellular carcinoma (liver cancer) or other cancers. No instances of thromboembolic events or cancers associated with Roctavian were observed in clinical studies. 

This application received Orphan, Breakthrough Therapy, Regenerative Medicine Advanced Therapy and Priority Review designations. 

Roctavian is approved with the AAV5 DetectCDx.

FDA release