Jan 9, 2020

BioMarin recently announced updates on their ongoing clinical trial program for their investigational gene therapy valoctocogene roxaparvovec, which was developed for the treatment of adults with severe hemophilia A. The therapy utilizes adeno-associated viruses (AAVs) as vectors to carry the genetic codes that prompt the production of the factor VIII (FVIII) protein that is deficient in people with hemophilia A. AAVs are designed to safely deliver the genetic material into living cells to sustained therapeutic effect.

The updates, which were recently published in the New England Journal of Medicine (NEJM), encompass three years of data from an ongoing Phase 1/2 study to evaluate safety and efficacy of valoctocogene roxaparvovec. Included in the trial are 15 participants with hemophilia A who are now in long term observation following a one-time administration of the therapy.  

According to a BioMarin press release, participants experienced significant improvements in both annualized bleeding rate (ABR) and requisite FVIII replacement product usage. In one group of patients who received a larger dose of the therapy, ABR decreased by 96%. In addition, FVIII product usage in the large dose group demonstrated sustained efficacy three years post-administration.

“These data are critical in helping the scientific and medical communities understand this pioneering technology. With three years of data, we know more about valoctocogene roxaparvovec than any other gene therapies in development for hemophilia A,” said Professor John Pasi, MB, ChB, PhD, from Barts and the London School of Medicine and Dentistry; the lead author of this NEJM publication, chief investigator for the valoctocogene roxaparvovec Phase 1/2 study, and a principal investigator for the Phase 3 study. “As a treating physician, I am excited about the potential of the field of gene therapy to make a meaningful difference in the lives of people with hemophilia A.”

The article, “Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A”
was published online in NEJM on January 2, 2020.

On December 23rd, the company submitted a Biologics License Application for valoctocogene roxaparvovec to the U.S. Food and Drug Administration.

Source: BioMarin press release dated January 2, 2020