BioPharma Dive June 30, 2023
Unexpected aspects of the hemophilia treatment’s prescribing information had some investors worried about its commercial prospects.
BioMarin Pharmaceutical, having secured a long-awaited approval from the Food and Drug Administration, says its focus has now turned to launching its new hemophilia gene therapy in the U.S. market.
The FDA on Thursday approved the therapy, known as Roctavian, for certain people with hemophilia A, the more common form of the rare bleeding disorder. While there are other effective treatments already available, the hope surrounding Roctavian has been that, at least for some patients, it could be a one-time fix for their disease.
For BioMarin, the launch will have a significant affect on its financial future. The company has already brought seven other...
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