A FVIII gene therapy treatment, developed by world leading gene therapy expert, Professor Amit Nathwani at UCL has shown positive results in treating patients with severe haemophilia A in a recent Biomarin sponsored Guys and St Thomas NHS Foundation Trust clinical trial. The FVIII gene therapy was licensed to BioMarin in 2013, they have recently applied for marketing authorisation with the EMEA and FDA.
The New England Journal of Medicine (NEJM) published an independently peer-reviewed article on up to three years of data from an ongoing Phase 1/2 study to evaluate safety and efficacy of investigational AAV gene therapy, valoctocogene roxaparvovec, for severe haemophilia A.
The data shows that all participating patients were able to stop their regular injections of clotting products due to the changes in their protein levels achieved by the gene therapy treatment.
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.