Struggling to find new patients, BioMarin Pharmaceuticals on Monday said it would restrict sales of its gene therapy for hemophilia A to three countries where it is currently approved, including the United States — a restructuring intended to reduce costs and help the treatment become profitable by next year.
The one-time treatment called Roctavian was approved in Europe in 2022 and in the U.S. in 2023. But in the second quarter, the company treated only five patients, generating $7 million in sales.
Under the new marketing plan, BioMarin will sell Roctavian in the U.S., Germany, and Italy, where it is currently approved and reimbursed. The company anticipates reducing Roctavian-related expenses to $60 million per year by 2025 and for the treatment to become profitable by the end of 2025.
