BioMarin looks to divest Roctavian gene therapy

BioMarin Pharmaceutical will pursue options to divest its hemophilia A gene therapy, Rocktavian, as part of a strategic alignment.

According to BioMarin, which made the announcement along with the company’s third quarter financial results, the decision to evaluate out-licensing options offers the most promising opportunity for ensuring continued patient access to Rocktavian.

The therapy, which employs an adeno-associated virus (AAV) vector to deliver a functional copy of the factor VIII gene to the liver, was approved in the U.S. in 2023 and carried a list price of approximately $2.9 million. It was heralded as groundbreaking for its potential to serve as a one-time treatment, eliminating the need for lifelong injections to replace the missing factor VIII.

But the high-priced drug’s commercial launch into a competitive hemophilia market was clouded by reimbursement hurdles. Struggling with a slow uptake of the therapy, in August 2024, BioMarin shifted its business strategy to focus commercial operations for Roctavian on three markets in high-income countries — U.S., Germany and Italy — where the drug had managed to secure reimbursement. 

Now, BioMarin plans to continue to make Rocktavian commercially available in the U.S., Italy and Germany until next steps are finalized. The company will also continue to provide support and monitoring for people treated with the therapy.