BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) has announced positive findings from ongoing worldwide third phase GENEr8-1 trial of valoctocogene roxaparvovec, experimental gene therapy for severe hemophilia treatment. With 134 participants, this is the world’s largest Phase 3 study for any gene treatment in hemophilia to date.
Annualized Bleeding Rate reduced by 85%
In the pre-specified interim analysis on subjects from a past non-interventional trial, the Annualized Bleeding Rate (ABR) was reduced significantly by 4.1 treated bleeds per year or 85% from a baseline mean of 4.8 (median 2.8). Throughout the efficacy evaluation period, the mean ABR was 0.8, while in year one, it was 0.9 (median 0.0), and during year two, the mean ABR was 0.7 (median 0.0).
Also, valoctocogene roxaparvovec considerably reduced the mean annualized Factor VIII infusion rate in the rollover group by 133 infusions annually or by 98% from baseline. Interestingly annualized infusion rate throughout the efficacy evaluation period was 2.6 (median 0.0), and during year one, it was 1.5 (median 0.0), while in year two mean annualized infusion rate was 3.4.
Study investigator Steven W. Pipe said, “A potential single treatment that provides a durable response for years could be a game-changer by offering a transformative treatment choice beyond existing therapies and addressing an unmet medical need for people with hemophilia A. As a principal investigator, I have witnessed the transformative liberating potential of valoctocogene roxaparvovec for hemophilia A in my own clinical trial participants. I’m delighted to see these results broadly confirmed in the largest study of its kind.”
Valoctocogene roxaparvovec could offer an alternative treatment to hemophilia A
BioMarin global R&D President Hank Fuchs said, “We are delighted that our perseverance on behalf of people with hemophilia A has led to today’s transformative results in the largest gene therapy study for hemophilia A. We are grateful for the support of the bleeding disorders community to conduct this clinical program. These results show that valoctocogene roxaparvovec could profoundly change the way hemophilia A is treated. We are looking forward to continuing to work with health authorities to bring this therapy to patients with hemophilia A.”