BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) has released additional data from its open-label Phase 1/2 study of a gene therapy called valoctocogene roxaparvovec aimed at treating severe hemophilia A.
The company had previously released a 4-year update of the results from the Phase 1/2 study of the investigative gene therapy. Professor John Pasi presented the recent data through an oral presentation on behalf of BioMarin during a virtual summit held by the World Federation of Hemophilia (WFH). The professor was the Chief investigator for the valoctocogene roxaparvovec Phase 1/2 study.
“With four years of data, this study represents the longest duration of clinical experience for any gene therapy in hemophilia A,” stated Professor Pasi.
Professor Pasi also pointed out that valoctocogene roxaparvovec has the potential to bring a paradigm shift in hemophilia A patients. This is because gene therapy presents an entirely new approach through which the unmet needs of patients with severe hemophilia A can be addressed.
BioMarin is eager to help improve the lives of severe hemophilia A patients
Hank Fuchs, BioMarin’s President of Global Research and Development, also stated that the company is committed to addressing bleeding disorders. It is also committed to helping the community of people suffering from those disorders, and especially severe hemophilia A through gene therapy. He also added that the company was pleased to share the data from the Phase 1/2 study of valoctocogene roxaparvovec with the WFH.
Fuchs also added that the study demonstrated a 96% decline in the sage of exogenous Factor VIII. This is because the treatment helps patients to generate endogenous factor VIII within their bodies, and this is a huge benefit to patients suffering from severe hemophilia A.
The company executive also revealed that BioMarin is working closely with regulators in different markets to secure approval that will enable the sale of the treatment in markets across the world. BioMarin expects the favorable results from the Phase 1/2 study of valoctocogene roxaparvovec will help sway regulatory decisions in its favor. The company is confident that they are on the right track with gene therapy.
