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 P-III GENEr8-1 study results involves assessing of valoctocogene roxaparvovec (6e13 vg/kg) in 20 patients with hemophilia A
  • The P-III GENEr8-1 study results: @23-26eks. 8 patients achieved Factor VIII levels of 40 IU/dL, mean ABR (1.5); reduction in median annualized Factor VIII usage (84%); reduction in mean FVIII usage (94%); mean & median Factor VIII level using the CS assay (36 IU/dL & 33 IU/dL)
  • Valoctocogene roxaparvovec is gene therapy developed utilizing an AAV-factor VIII vector and has received FDA’s BT designation, EMA’s PRIME designation for hemophilia A with its expected FDA & EU regulatory submission in Q3’19

Click here to read full press release/ article | Ref: BioMarin | Image: BioMarin

Tuba Khan

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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