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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Coming year could feature first-time approvals in the EU for at least five gene therapies.
The company plans to file with 52-week data for AMT-061 and is confident it won't face the same stringent requirements from the FDA that led to a complete response letter for BioMarin's Roctavian.