February 3, 2023

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) – BioMarin’s Hemophilia Gene Therapy Meets Efficacy Endpoints At Two Year Analysis


BioMarin Pharmaceutical Inc (NASDAQ: BMRN) announced results from its ongoing Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, investigational gene therapy for severe hemophilia A. 

  • Annualized Bleeding Rate (ABR) was significantly reduced by 4.1 treated bleeds per year in the pre-specified primary analysis in participants from a prior non-interventional study.
  • Valoctocogene roxaparvovec also significantly reduced the mean annualized Factor VIII infusion rate in the rollover population by 133 infusions per year or 98% from baseline. 
  • Related: Five-Year Data Of BioMarin’s Hemophilia Gene Therapy Shows Sustained Benefit.
  • Overall, valoctocogene roxaparvovec has continued to be well tolerated. 
  • No participants developed inhibitors to Factor VIII, malignancy, or thromboembolic events.
  • The European Medicines Agency validated BioMarin’s resubmission of a marketing application. A Committee for Medicinal Products for Human Use and Committee for Advanced Therapies opinion is anticipated in 1H of 2022. 
  • In the U.S., BioMarin intends to submit two-year follow-up safety and efficacy data GENEr8-1 study to support the benefit/risk assessment of valoctocogene roxaparvovec, as previously requested by the FDA.
  • The Company plans to meet with FDA to discuss the resubmission of a marketing application targeted for Q2 2022, followed by an expected six-month review by the FDA.
  • Price Action: BMRN shares are down 1.17% at $84.25 during the premarket session on the last check Monday.



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