BioMarin Delays Resubmission, But Roctavian’s Rocky Road Winding Down
FDA Request Means Company Expects BLA By End Of September
The company had planned to refile its application at the end of June, though it is gearing up for potential approval and commercialization in Europe that could provide important experience for the US.
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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 company plans to start in Germany with an outcomes-based payment model. But while a US FDA nod seems likely, analysts have been more cautious about calling US approval a done deal.
Will BioMarin be second time lucky with its hemophilia gene therapy Roctavian in the EU? The European Medicines Agency could soon decide whether the product should be approved.