BioMarin Shares Additional Promising Phase I/II Data on Hemophilia A Treatment

BioMarin HQ/ Courtesy of BioMarin

BioMarin HQ/courtesy of BioMarin

California-based BioMarin Pharmaceutical released new data today from its previously reported four-year update of the open-label Phase I/II study of valoctocogene roxaparvovec. The investigational gene therapy treatment is designed for patients with severe hemophilia A. The results were presented at the World Federation of Hemophilia (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.

In the six study participants who were previously on Factor VIII prophylaxis in the 6e13 vg/kg cohort, the data showed substantial, sustained reductions in bleeding.

"With four years of data, this study represents the longest duration of clinical experience for any gene therapy in hemophilia A,” said Professor Pasi. “It is exciting to observe that all study participants remain off Factor VIII prophylaxis therapy, while also experiencing a greater than 90 percent reduction in bleeding episodes from a single administration of valoctocogene roxaparvovec. These data demonstrate the very real potential of a paradigm shift in the treatment of hemophilia 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 hemophilia A."

The safety profile of valoctocogene roxaparvovec remains consistent with previously reported data. There were no reported delayed-onset, treatment-related events. The global Phase III study of valoctocogene roxaparvovec at the 6e13 vg/kg dose is looking into the superiority of the product compared to current standards of care.

"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 hemophilia A," said Hank Fuchs, M.D., President, Global Research and Development at BioMarin.

Progress has been made in the treatment realm for hemophilia A as of late. Expression Therapeutics announced at the end of May that it had received clearance from the U.S. Food and Drug Administration to proceed with its Investigational New Drug Application for the clinical testing of its novel lentiviral vector-based gene therapy, ET3.

Following this news, Expression Therapeutics specified that it expects to initiate a Phase I clinical trial titled ET3-201 to look into ET3 as a potential treatment for hemophilia A in the near future.

"We are very excited to get the hemophilia A clinical trial underway, the first of six gene therapy products currently under development at Expression Therapeutics," said Mohan Rao, Ph.D., CEO of Expression Therapeutics.

Takeda Pharmaceutical Company Limited also announced updates on June 15 from its AHEAD global study, examining the long-term outcomes associated with ADVATE (antihemophilic factor [recombinant], rAHF) in patients with hemophilia A.

“The results of the interim analysis from the AHEAD real-world outcomes study support the long-term effectiveness of rAHF for hemophilia A patients, which was maintained in a six-year observational period and bolstered by consistent [health-related quality-of-life] scores,” said Prof. Dr. med. Johannes Oldenburg, Chairman and Director, Institute of Experimental Haematology and Transfusion Medicine, University Clinic Bonn AöR, Germany, who is the researcher leading the study. “Every patient is unique, and these results support a tailored and personalized approach to optimize care for patients with bleeding disorders, delivered in partnership with their treating physician.”

Hemophilia is a chronic disease that causes longer-than-normal bleeding due to absent or deficient clotting factor within the blood. In 2018, hemophilia A impacted more than 173,000 people worldwide.

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