BioMarin Reports Submission of MAA to EMA for Valoctocogene Roxaparvovec to Treat Severe Hemophilia A

BioMarin Reports Submission of MAA to EMA for Valoctocogene Roxaparvovec to Treat Severe Hemophilia A

Shots:

  • The MAA submission is based on ongoing P-III study with material from the to-be-commercialized process and updated 3yrs. P-I/II data. The submission marks the first MAA submission for a gene therapy product targeting any type of hemophilia
  • BioMarin is expected its MAA for Valoctocogene Roxaparvovec to be reviewed under expedited accelerated assessment designation in Jan’2020
  • Valoctocogene roxaparvovec is gene therapy developed utilizing an AAV-factor VIII vector and has received FDA’s BT designation, EMA & FDA’s ODD for hemophilia A with its expected BLA submission to the US FDA by the year-end

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