FDA rejects BioMarin's gene therapy and Gilead's filgotinib over concerns
The FDA refused to approve BioMarin's hemophilia A gene therapy valoctocogene roxaparvovec. The U.S. regulator that had earlier told there was no requirement of an AdComm for the drug, aka, valrox, has issued a complete response (CRL) letter, which could cause a significant delay for any future approval, and canceled what would have been the first-ever approval for gene therapy in the bleeding disorder.
The removal of an AdComm was seen as a positive. Three-year data on its candidate were reported last year, but it sparked concerns regarding the durability of the therapy after factor VIII ...
