BioMarin Reports Results of Valoctocogene Roxaparvovec in P-III GENEr8-1 Study for Hemophilia A

 BioMarin Reports Results of Valoctocogene Roxaparvovec in P-III GENEr8-1 Study for Hemophilia A

BioMarin Reports Results of Valoctocogene Roxaparvovec in P-III GENEr8-1 Study for Hemophilia A

Shots:

  • The ongoing P-III GENEr8-1 study involves assessing of valoctocogene roxaparvovec (6e13 vg/kg dose) in 134 patients with with severe hemophilia A. The patients received a single dose of valoctocogene roxaparvovec and completed a year or more of follow-up
  • The study met 1EPs & 2EPs i.e. reduction in ABR by 84% demonstrating superiority to factor VIII prophylaxis and reduced mean annualized factor VIII infusion rate by 99%. In subset dosed >2yrs. ago, rate of decline in factor VIII expression is slow
  • Mean ABR in the population was 0.9 over these two-plus yrs. BioMarin plans to meet with EMA to discuss the submission of 1yrs. data and the FDA to review 2yrs. data-request

Click here ­to­ read full press release/ article | Ref: PRNewswire | Image: Northbay Biz

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Tuba Khan is Senior Editor at PharmaShots. She is curious, creative, and passionate about recent updates and innovation in the Life sciences industry. She covers Biopharma, MedTech, and Digital health segments. Tuba also has an experience of digital and social media marketing and runs the campaigns independently. She can be contacted on tuba@pharmashots.com

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