Not Such A Sure Thing: FDA Knocks Back BioMarin’s Roctavian
Company’s Hemophilia A Gene Therapy Gets A CRL
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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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.
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.