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.