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image credit: The U.S. Food and Drug Administration / Wikimedia

FDA gets out its red pen again, rejecting BioMarin’s gene therapy valrox amid durability worries

August 19, 2020

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BioMarin’s shares plummeted in early Wednesday trading after the FDA refused to approve its hemophilia A gene therapy valoctocogene roxaparvovec.

The U.S. regulator, which had earlier said there was no need for an AdComm for the drug, also known as valrox, has issued a complete response (CRL) letter that could cause a major delay for any future approval, and nixed what would have been the first-ever approval for a gene therapy in the bleeding disorder.

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