FDA delays review of BioMarin's hemophilia gene therapy after data update
The FDA has extended its review of BioMarin’s gene therapy for hemophilia A by three months, formalizing an expected delay that will now push out a decision to the end of June.
BioMarin told investors in November, after submitting three-year data analysis from a Phase III trial, that the FDA said it may constitute a major amendment that warrants extra time for review.
And that’s exactly what happened, with the agency citing “substantial amount of additional data,”
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.
While the FDA had planned on convening an advisory committee, it ultimately decided not to, which analysts saw as a positive sign.
If approved, valrox would be the first FDA-approved gene therapy to treat severe hemophilia A by replacing the gene for the blood clotting protein factor VIII. CSL won approval for a hemophilia B gene therapy late last year.
In August, the European Commission granted conditional marketing authorization to valrox under the brand name Roctavian.