Posted by Michael Wonder on 28 Oct 2017
FDA grants breakthrough therapy designation for BioMarin's valoctocogene roxaparvovec (formerly BMN 270), an investigational gene therapy for hemophilia A
26 October 2017 - Global phase 3 program to begin before year end.
BioMarin Pharmaceutical announced today that the U.S. FDA granted valoctocogene roxaparvovec (formerly BMN 270) breakthrough therapy designation. The FDA's breakthrough therapy designation program is intended to facilitate and expedite development and review of new drugs to address unmet medical need in the treatment of a serious condition. To qualify for breakthrough therapy designation, preliminary clinical evidence must show that that the drug may demonstrate substantial improvement over existing therapies.
BioMarin expects to initiate enrolment of a global Phase 3 program before the end of the year.
