BioMarin Pharmaceuticals Inc. (NASDAQ: BMRN) has announced the publication of Phase 3 GENEr8-1 study results of valoctocogene roxaparvovec in the New England Journal of Medicine. Valoctocogene roxaparvovec is an experimental gene therapy for severe hemophilia treatment in adults.
BioMarin publishes GENEr8-1 Study results
The publication titled “Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A” offers one year plus follow-up findings from the trial and is referenced in an editorial published in the journal that acknowledges the advantages of zero bleeds and shunning the use of prophylactic therapy
Interestingly the initial research article reported that after a single valoctocogene roxaparvovec infusion, subjects demonstrated a considerable reduction in annualized bleeding rates, increased factor VII activity, and reduced factor VII utilization compared to before they were enrolled. Notably, in the pro-specified rollover sample comprising 112 subjects enrolled from a possible noninterventional study, average annualized factor VII concentrate user and average treated bleed rates at four weeks dropped 99% and 84%, respectively post-infusion.
In general, around 90% of the subjects had either fewer or no treated bleeds the following infusion, as indicated in the noninterventional study. After 49 to 52 weeks, 885 of the subjects had a median factor VIII activity of 5IU/dL or more as measured using Chromogenic substrate assay.
90% of subjects had zero bleeds after a year
Margareth Ozelo, the lead principal investigator of the GENE48-1 study, said, “Breakthrough bleeding represents a high burden of disease management and an unmet medical need for many people. I am encouraged that during the first year of treatment, 90% of study participants had either zero treated bleeds or fewer treated bleeds post-infusion than with factor VIII prophylaxis.”
BioMarin Global Research and Development President Hank Fuchs said, “Valoctocogene roxaparvovec has been studied longer than any other gene therapy for hemophilia A, and year after year, we continue to increase our knowledge of how this investigational therapy may potentially benefit the lives of people with hemophilia A. We are grateful to the study participants and investigators for their essential role in this development program, which includes GENEr8-1, the largest gene therapy study in hemophilia A.”