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.
