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FDA Grants Breakthrough Therapy Designation for BioMarin’s Investigative Hemophilia A Gene Therapy

October 30, 2017
Hemophilia A Gene Therapy Gets Breakthrough Status

The U.S. Food and Drug Administration (FDA) recently granted “breakthrough” therapy designation for valoctocogene roxaparvovec (formerly BMN 270), an investigational hemophilia A gene therapy candidate developed by BioMarin Pharmaceuticals.

The breakthrough status was granted in light of positive results of phase 1/2 clinical study that focused on the safety and efficacy of valoctocogene roxaparvovec. Breakthrough drugs are put on a fast-track approval program and given intensive guidance from the FDA. The therapy was granted a similar designation by the European Medicines Agency (EMA), the FDA’s counterpart in Europe, earlier in 2017.  

Valoctocogene roxaparvovec uses adeno-associated viruses (AAVs) as delivery vehicles, or vectors, to carry the genetic codes that prompt the production of the factor VIII (FVIII) protein that is deficient in people with hemophilia A. These AAVs are designed to deliver genetic material into living cells to sustain therapeutic effect without causing disease or triggering significant immune responses.

“The news of the FDA granting breakthrough therapy designation coupled with EU PRIME designation granted in early 2017 by EMA, demonstrates the strong support of global health authorities for valoctocogene roxaparvovec and its expedited development and registration pathway,” said Hank Fuchs, MD, President, Worldwide Research and Development at BioMarin. “There is a tremendous need to achieve normal steady state factor VIII levels to eliminate spontaneous bleeding, to avoid the complications of sub optimally corrected bleeding disorder, to improve quality of life and enable patients to live to their fullest potential.”

BioMarin has indicated that patient enrollment for phase 3 clinical studies will begin before the end of 2017.

Source: BioMarin press release dated October 26, 2017