First gene therapy to treat severe haemophilia A

EMA

24 June 2022 - EMA has recommended granting a conditional marketing authorisation in the European Union (EU) for Roctavian (valoctocogene roxaparvovec) for the treatment of severe haemophilia A in adults who do not have factor VIII inhibitors (auto-antibodies produced by the immune system which make factor VIII medicines less effective) and no antibodies to adeno-associated virus serotype 5 (AAV5).

EMA’s recommendation is based on the results of a Phase 3 single-arm (main study), non-randomised study in 134 male patients with haemophilia A without a history of factor VIII inhibitor and without detectable pre-existing antibodies to AAV5.

Read EMA press release 

Michael Wonder

Posted by:

Michael Wonder

Posted in:

Outcome , Europe , Gene therapy