BioMarin’s Gene Therapy for Hemophilia Gets Extended Review by FDA

March 8, 2023

BioMarin will have to face another delay on the review of its hemophilia A review by the US Food and Drug Administration. This comes after the company shared data from ongoing clinical trials in November of last year. The drug was previously rejected, but Biomarin’s analysts are optimistic about the drug’s odds based on the new data.

According to , “It’s been almost three years since BioMarin was slammed with the FDA’s surprise rejection of its then-top program, valoctocogene roxaparvovec or valrox. While the biotech had filed for approval with data from a Phase I/II study and preliminary updates from a Phase III, regulators demanded to see two years of follow-on data from that Phase III trial to prove that valrox can stem bleeds durably. The company has since put together that data package, with promises to offer even more. In addition to the three-year data it now has on hand, BioMarin also proposed a long-term extension study and other post-approval registry studies.”

To read more, click here.