STAT August 5, 2024
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...