BioMarin to restrict hemophilia therapy to three countries, seeking to overcome slow sales

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.

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