BioMarin Prepares Investors For A Possible US Hemophilia Gene Therapy Delay
FDA review of the company's BLA for a hemophilia gene therapy could be delayed by three months to allow the agency to review a three-year data analysis from an ongoing trial.
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Peter Marks says labeling for CSL’s hemophilia B treatment adequately conveys its risks. He also denies NHF request that the label cite eligibility criteria used in clinical trials, responding to a citizen petition on the day the agency approves the therapy.
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.
The approval is a breakthrough for BioMarin, which is hoping to seize market share from Factor VIII replacement therapies and Roche’s Hemlibra.