FDA aknowledged hemophilia gene therapy as Breakthrough Designation

Print 31 October 2017
GMP News

BioMarin Pharmaceutical Inc. announced today 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.

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.

“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, 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.”

The European Medicines Agency (EMA) has granted access to its Priority Medicines (PRIME) regulatory initiative for valoctocogene roxaparvovec. To be accepted into PRIME, an investigational therapy has to show its potential to benefit patients with unmet medical needs based on early clinical data. PRIME focuses on medicines that may offer a major therapeutic advantage over existing treatments, or benefit patients with no treatment options. These medicines are considered priority medicines within the European Union (EU).

BioMarin’s valoctocogene roxaparvovec has also received orphan drug designation from the FDA and EMA for the treatment of hemophilia A. The Orphan Drug Designation program is intended to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions.

BioMarin has constructed one of the largest gene therapy manufacturing facilities in the world, which is located in Novato, California, USA. Good Manufacturing Practices (GMP) production of valoctocogene roxaparvovec has commenced and will support clinical development activities and anticipated commercial demand.

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