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Home » BioMarin Faces New FDA Hurdle for Its Highly-Anticipated Hemophilia Treatment
BioMarin Faces New FDA Hurdle for Its Highly-Anticipated Hemophilia Treatment
BioMarin Pharmaceutical announced that the FDA has declined to approve its investigational gene therapy for severe hemophilia A, valoctocogene roxaparvovec, and the agency wants to see two years of follow-up data from a phase 3 study.