BioMarin Pharmaceutical announced that the U.S. Food and Drug Administration (FDA) granted valoctocogene roxaparvovec (formerly BMN 270) Breakthrough Therapy Designation. The FDA’s Breakthrough Therapy Designation program is intended to facilitate and expedite development and review of new drugs to address unmet medical need in the treatment of a serious condition. To qualify for Breakthrough Therapy Designation, preliminary clinical evidence must show that that the drug may demonstrate substantial improvement over existing therapies. BioMarin expects to initiate enrollment of a global Phase 3 program before the end of the year.
“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, M.D., 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 suboptimally corrected bleeding disorder, to improve quality of life and enable patients to live to their fullest potential.”
Breakthrough Therapy Designation status was granted based on the data from an ongoing BioMarin Phase 1/2 study, which evaluated safety and efficacy of valoctocogene roxaparvovec. Interim results of the study were provided at the company’s R&D Day for investors last week.
The global Phase 3 program includes two studies with valoctocogene roxaparvovec, one with the 4e13 vg/kg dose and one with the 6e13 vg/kg dose. The studies will each likely include approximately 40 patients. In addition, the Company has commissioned its commercial gene therapy manufacturing facility to supply both clinical and commercial drug.