BioMarin to pull Roctavian gene therapy from market

Despite comprehensive yet unsuccessful efforts to identify a potential buyer, BioMarin will voluntarily withdraw its Roctavian gene therapy from the market.

Back in October 2025, BioMarin revealed that it was pursuing options to divest the gene therapy, as part of a strategic alignment.

Unable to find a buyer, the company's strategic decision to pull Roctavian from the market has resulted in charges of approximately $240 million during Q4 of 2025, including a $119 million inventory write-off and $118 million in asset impairments, BioMarin noted in its most recent financial report.

Roctavian, 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. for severe hemophilia A 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.