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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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.
BioMarin Rolls Up Sleeves To Prepare For Roctavian’s EU Debut
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.
BioMarin’s Roctavian & Argenx’s Efgartigimod Among Eight Hopefuls Awaiting EU Verdict
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.