Development of new treatment for severe haemophilia A on track

21 November 2019


BioMarin today announced it has submitted a marketing application to the European Medicines Agency (EMA), for the investigational gene therapy, valoctocogene roxaparvovec. BioMarin is also on track to submit a marketing application to the United States health authorities by the end of this year.

The gene therapy was originally licensed to BioMarin by UCLB based on research from the laboratory of Professor Amit Nathwani and his team at UCL and collaborating with researchers at St. Jude Children’s Research Hospital.

“Valoctocogene roxaparvovec shows a transformational reduction in bleeding and need for factor VIII infusions…” said Hank Fuchs, M.D., President Worldwide Research and Development at BioMarin.

About BioMarin and Disease Information

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases.  The Company’s portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates.  For additional information, please visit www.biomarin.com.  Information on such website is not incorporated by reference into this press release.

About Haemophilia A

Haemophilia A, also called factor VIII (FVIII) deficiency or classic haemophilia, is a genetic disorder caused by missing or defective factor VIII, a clotting protein. Although it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people is born with Haemophilia A. People living with the disease are not able to form blood clots efficiently and are at risk for excessive bleeding from modest injuries, potentially endangering their lives. People with severe haemophilia often bleed spontaneously into their muscles or joints. The standard of care for the 43% of individuals with haemophilia A who are severely affected is a prophylactic regimen of Factor VIII infusions two to three times per week. Even with prophylactic regimens, many people still experience spontaneous bleeding events that result in progressive and debilitating joint damage.

To read the full BioMarin story click here:

Poised for Significant Growth and Profitability, BioMarin Shares Company Highlights During R&D Day on November 14th in New York  

BioMarin Submits Marketing Authorization Application to European Medicines Agency for Valoctocogene Roxaparvovec to Treat Severe Haemophilia A