BioMarin Receives the US FDA’s Priority Review Acceptance of Valoctocogene Roxaparvovec’s BLA for Patients with Hemophilia A

Shots:

• The FDA acceptance marks the first marketing application accepted for a gene therapy to treat hemophilia in the US. The application is based on ongoing P-III study with material from the to-be-commercialized process and updated 3yrs. P-I/II data
• Additionally, the FDA has accepted the PMA for an AAV5 total antibody assay intended as CDx for valoctocogene roxaparvovec. The US FDA stated that they not planning to hold an advisory committee meeting to review the application
• 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 anticipated PDUFA date as Aug 21, 2020

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