10 November 2017

BioMarin today announced that the U.S. Food and Drug Administration (FDA) granted valoctocogene roxaparvovec (formally BMN 270) a Breakthrough Therapy Designation for the treatment of hemophilia A.

BMN 270 gene therapy for severe haemophilia A was originally licensed by UCLB based on research from the laboratory of Professor Amit Nathwani and his team at UCL in collaboration with researchers at St. Jude Children’s Research Hospital.

To qualify for Breakthrough Therapy Designation, preliminary clinical evidence must show that that the drug may demonstrate substantial improvement over existing therapies.

Read the full story here: http://investors.biomarin.com/2017-10-26-FDA-Grants-Breakthrough-Therapy-Designation-for-BioMarins-Valoctocogene-Roxaparvovec-formerly-BMN-270-an-Investigational-Gene-Therapy-for-Hemophilia-A