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BioMarin Reports Positive Phase 3 Gene Therapy Trial Results In Adults With Severe Hemophilia A

BioMarin Pharmaceutical Inc. (BMRN) announced positive phase 3 gene therapy trial results in adults with Severe Hemophilia A. The study met all primary and secondary efficacy endpoints in one-year data set.

Data from the GENEr8-1 phase 3 study with a mean follow-up of 71.6 weeks showed that in the pre-specified primary analysis for annualized bleeding rate a single dose of valoctocogene roxaparvovec significantly reduced annualized bleeding rate by 84%. 80% of participants were bleed-free starting at week five after treatment.

The company noted that Valoctocogene roxaparvovec also significantly reduced the mean annualized Factor VIII in the rollover population by 99% from 135.9 to 2.0 infusions per year.

At the end of the first year post-infusion with valoctocogene roxaparvovec, participants in the modified intent-to-treat or mITT population had a mean endogenous Factor VIII expression level of 42.9 IU/dL, as measured by the chromogenic substrate assay, supporting the marked clinical benefits observed with abrogation of bleeding episodes and Factor VIII infusion rate.

Factor VIII expression declined at a slower rate compared to the Phase 1/2 study, and remained in a range to provide hemostatic efficacy.

In a subset of the mITT population that had been dosed at least two years prior to the data cut date, Factor VIII expression declined from a mean of 42.2 IU/dL at the end of year one to a mean of 24.4 IU/dL at the end of year two with continued hemostatic efficacy demonstrated by a mean annualized bleeding rate of 0.9 bleeding episodes per year.

Overall, in the Phase 3 study, valoctocogene roxaparvovec has been well tolerated by the 134 participants who received a single 6e13 vg/kg dose. No participants developed inhibitors to Factor VIII, or thromboembolic events. One participant was lost to follow-up. Infusion-related reactions were effectively mitigated by managing infusion rates, the company said.

BioMarin said it is working with the U.S. Food and Drug Administration to align on steps forward to obtain marketing approval for valoctocogene roxaparvovec gene therapy for severe hemophilia A. The FDA recommended that the company complete the Phase 3 study and submit two-year follow-up safety and efficacy data on all study participants.

Inddition, the European Medicines Agency (EMA) requested one-year results from the full Phase 3 study to inform their benefit-risk assessment. BioMarin plans to resubmit the MAA with these data to the EMA in the second quarter of 2021 following discussions with the Agency.

The FDA has granted valoctocogene roxaparvovec Breakthrough Therapy Designation. BioMarin's valoctocogene roxaparvovec has 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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