BioMarin Provides 1.5 years of Clinical Data for Valoctocogene Roxaparvovec Gene Therapy for Severe Haemophilia A

15 December 2017


Valoctocogene Roxaparvovec Demonstrated Sustained Normal or Near Normal Factor VIII Levels in Severe Haemophilia A for Most Patients at Both Doses in Phase 1/2 Study.

BioMarin Pharmaceutical Inc. have announced an update to its previously reported results of an open-label Phase 1/2 study of valoctocogene roxaparvovec (formerly BMN 270), an investigational gene therapy treatment for severe haemophilia 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.

The updated results were presented at the 59th American Society of Hematology (ASH) Annual Meeting and Exposition by John Pasi, M.B., Ch.B., Ph.D., from Barts and the London School of Medicine and Dentistry and primary investigator for this Phase 1/2 study. Data presented illustrates sustained normal or near-normal Factor VIII levels in severe haemophilia A for most patients taking part.

Read the full story here: http://investors.biomarin.com/2017-12-09-BioMarin-Provides-1-5-years-of-Clinical-Data-for-Valoctocogene-Roxaparvovec-Gene-Therapy-for-Severe-Hemophilia-A-at-59th-American-Society-of-Hematology-ASH-Annual-Meeting-Concurrent-with-NEJM-Publication?printable


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. As an X-linked disorder, haemophilia A mostly affects males, occurring in approximately 1 in 5,000 male births. 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 life. People with severe haemophilia often bleed spontaneously into their muscles or joints. The standard of care for the 43% of haemophilia A patients who are severely affected, is a prophylactic regimen of Factor VIII infusions three times per week. Even with prophylactic regimens, many patients still experience microbleeds and spontaneous bleeding events that result in progressive joint damage.

About BioMarin
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 BioMarin’s website is not incorporated by reference into this press release.

About UCL Business PLC
UCL Business PLC (UCLB) is a leading technology commercialisation company that supports research and innovations arising from UCL, one of the UK’s top research-led universities. UCLB has a successful track record and a strong reputation for identifying and protecting promising new technologies and innovations from UCL academics. UCLB has a strong track record in commercialising medical technologies and provides technology transfer services to UCL’s associated hospitals; University College London Hospitals, Moorfields Eye Hospital, Great Ormond Street Hospital for Children and the Royal Free London Hospital. It invests directly in development projects to maximise the potential of the research and manages the commercialisation process of technologies from laboratory to market.
For further information, please visit: www.uclb.com