It priced the one-time therapy, Roctavian, at $2.9 million. BioMarin said most study participants continued to respond to the gene therapy through year three and beyond, and it would continue to monitor them for 15 years. BioMarin's therapy will compete for market share with Roche's (ROG.S) Hemlibra, an antibody drug which mimics the function of the blood clotting protein missing in hemophilia A patients. In April, BioMarin cut its annual sales forecast range for Roctavian to $50 million to $100 million, from $100 million to $200 million. There are about 16,000 patients in the United States with hemophilia A in which they are missing the factor VIII clotting protein.

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Persons: BioMarin, Joel Beatty, Robert W Baird, Roctavian, Beatty, Khushi Mandowara, Bhanvi Satija, Nathan Gomes, Akash Sriram, Deena Beasley, Krishna Chandra Eluri, Maju Samuel, David Gregorio Our Organizations: U.S . Food, Drug Administration, European Union, Los, Thomson Locations: U.S, California, Novato , California, hemophilia, United States, Bengaluru, Los Angeles