BioMarin reveals new data for haemophilia gene therapy

Biotech News: BioMarin announced new data from its phase 1/2 study of valoctocogene roxaparvovec, an investigational gene therapy treatment for severe haemophilia A.

The results were presented during a late-breaking oral presentation at the World Federation of Haemophilia (WFH) Virtual Summit by Professor John Pasi, M.B., Ch.B., Ph.D., from Barts and the London School of Medicine and Dentistry and Chief Investigator for this Phase 1/2 study. 

“With four years of data, this study represents the longest duration of clinical experience for any gene therapy in haemophilia A.  It is exciting to observe that all study participants remain off Factor VIII prophylaxis therapy, while also experiencing a greater than 90% reduction in bleeding episodes from a single administration of valoctocogene roxaparvovec,” said Professor Pasi. “These data demonstrate the very real potential of a paradigm shift in the treatment of haemophilia A and that ongoing research into gene therapies could represent an entirely new way to approach meeting the high unmet need in patients with severe haemophilia A.”

“BioMarin is committed to the bleeding disorders community with the most robust and advanced clinical development program for a potential first gene therapy in severe haemophilia A,” said Hank Fuchs, M.D., President, Global Research and Development at BioMarin. “We are pleased to share these data at WFH.  Demonstrating a 96% reduction in exogenous Factor VIII usage as patients are now producing their own endogenous factor VIII is a potential benefit that we hope to be able to offer as we work closely with regulators to seek approval and work to reduce the burden of haemophilia.”

The data presented at WFH is the most current data (April 8, 2020, cut off) and includes four years of data for the 6e13 vg/kg cohort and three years of data for the 4e13 vg/kg cohort.