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Not Such A Sure Thing: FDA Knocks Back BioMarin’s Roctavian

Company’s Hemophilia A Gene Therapy Gets A CRL

Executive Summary

BioMarin’s confidence that its first-ever hemophilia gene therapy would be cleared by the US FDA was shaken by a complete response letter that the company claims changed the requirements for approval.

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Round Two: BioMarin Resubmits Hemophilia A Gene Therapy Roctavian In US

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 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.

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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.

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