BioMarin Pharmaceutical Inc., despite multiple setbacks, easily maintained its lead in the race to bring a hemophilia A gene therapy to market in the US with the resubmission of its biologic license application for Roctavian (valoctocogene roxaparvovec), kicking off a review process that could result in US Food and Drug Administration approval by mid-2023.
Round Two: BioMarin Resubmits Hemophilia A Gene Therapy Roctavian In US
Conditional Approval Granted In EU In August
BioMarin maintains its lead in the race to win approval for a hemophilia A gene therapy in the US by resubmitting Roctavian two years after the FDA issued a CRL in response to the original BLA.
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The promise of innovative therapies seems to have been constrained not by efficacy or safety concerns, but because the high price of treatments is incongruous with the reimbursement of short-course therapies.
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