BioMarin Pharmaceutical announced its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A achieved pre-specified clinical criteria for regulatory review in the U.S. and Europe. As of May 28, 2019, eight patients in the 20-patient cohort of the Phase 3 GENEr8-1 study achieved Factor VIII levels of 40 international units per deciliter (IU/dL), or more, at 23 to 26 weeks, meeting the pre-specified criteria for Factor VIII activity levels. The company will meet with the Food and Drug Administration (FDA) and European Medicines Agency (EMA) to review the phase 3 data and the other elements of a submission and intends to announce the timing for its planned marketing applications in 3Q 2019.

As of the April 30, 2019 data cutoff, between weeks 23 to 26, in a cohort of the Phase 3 GENEr8-1 study of valoctocogene roxaparvovec dosed at 6e13 vg/kg, seven of 16 study participants reached or exceeded the pre-specified Factor VIII levels of 40 international units per deciliter using the chromogenic substrate (CS) assay. Subsequent to the April 30 cutoff, one additional participant met that criteria, bringing the total to eight participants.

For the 16 patients who had reached week 26 by the April 30 cutoff since administration of valoctocogene roxaparvovec, the estimated median Annual Bleed Rate (ABR) was zero and the estimated mean ABR was 1.5, representing a reduction of 85% from baseline levels where all patients were on standard of care prophylaxis. In addition, there was an 84% reduction in median annualized Factor VIII usage and a 94% reduction in mean FVIII usage annualized between week 5 and 26. In the 23 to 26 week time period the mean Factor VIII level using the CS assay was 36 (SD=28) IU/dL and the median was 33 IU/dL.

"Reaching this pre-specified clinical endpoint is an important milestone that brings us one step closer to a potential regulatory submission in both the U.S. and Europe for valoctocogene roxaparvovec to treat adults with severe hemophilia A," said Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin. "Our discussions with the FDA and EMA underscore the high level of unmet need in the hemophilia community, and we look forward to continuing our productive dialogue on the submissions."

The U.S. Food and Drug Administration (FDA) granted valoctocogene roxaparvovec Breakthrough Therapy Designation. The FDA's Breakthrough Therapy Designation program is intended to facilitate and expedite development and review of new drugs to address unmet medical need in the treatment of a serious condition. To qualify for Breakthrough Therapy Designation, preliminary clinical evidence must show that the drug may demonstrate substantial improvement over existing therapies.

The European Medicines Agency (EMA) has granted access to its Priority Medicines (PRIME) regulatory initiative for valoctocogene roxaparvovec. To be accepted for PRIME, an investigational therapy has to show its potential to benefit patients with unmet medical needs based on early clinical data. PRIME focuses on medicines that may offer a major therapeutic advantage over existing treatments, or benefit patients with no treatment options. These medicines are considered priority medicines within the European Union (EU).

BioMarin's valoctocogene roxaparvovec has also received orphan drug designation from the FDA and EMA for the treatment of severe hemophilia A. The Orphan Drug Designation program is intended to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions.

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