image credit: The U.S. FDA / Wikimedia

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

August 19, 2020


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.

Read More on FierceBiotech