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

Company’s Hemophilia A Gene Therapy Gets A CRL

 
• By Mandy Jackson

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.

Hand holds a drop of blood. World Hemophilia Day. World Blood Donor Day.
Patients may have to wait two years or more for the first hemophilia A gene therapy • Source: Shutterstock

BioMarin Pharmaceutical Inc. insists that it did not know that the US Food and Drug Administration wanted to see longer-term data on its gene therapy Roctavian (valoctocogene roxaparvovec) for severe hemophilia A until it received a complete response letter from the agency.

The CRL, announced 19 August and received a day earlier, was a complete surprise. BioMarin had been very confident, and...

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