FDA approves first gene therapy for hemophilia A

June 30, 2023

The U.S. FDA has approved BioMarin Pharmaceutical's Roctavian, the first and only gene therapy for adults with severe hemophilia A.

Roctavian gene therapy is approved for the treatment of adults with severe hemophilia A without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test.

The one-time, single-dose infusion was first approved by the EMA in August 2022. In the U.S., Roctavian's BLA was delayed twice — once in November 2022 and then again this past March.The most recent delay came after the FDA determined that the submission of the data analysis from an ongoing phase 3 study constituted a Major Amendment.

The FDA approval is based on data from the global phase 3 GENEr8-1 study. The 134-patient trial, according to California-based BioMarin, is the largest phase 3 trial of any gene therapy in hemophilia. The study showed a 50% reduction in annual bleeding incidents among treated patients.

Last year, the FDA approved the first gene therapy for adults with hemophilia B, a less common form of hemophilia — CSL Behring's Hemgenix — and it quickly became the world's most expensive drug, priced at $3.5 million for the one-time treatment. BioMarin has indicated that Rocktavian will be priced at $2.9 million.