Rare Disease Report

Genzyme's Oral Treatment for Fabry Disease Gets Fast Tracked by the FDA

APRIL 28, 2015
James Radke, PhD

The FDA has granted Fast Track designation for the development of  Genzyme’s GZ/SAR402671, an oral substrate reduction therapy being developed for the treatment of Fabry disease.

Fabry disease is an X-linked lysosomal storage disorder that leads to excessive deposition of globotriaosylceramide ( GL-3) throughout the body. Skin, eye, kidney, heart, brain, and peripheral nervous system are highly vulnerable. Currently, the only treatment available is agalsidase alfa (Fabrazyme) which is an enzyme replacement therapy infused over several hours every two weeks.

The Fast Track Designation for the oral GZ/SAR402671 provides Genzyme with frequent interactions with the FDA review team to expedite the clinical development and review of a New Drug Application (NDA). The Fast Track Designation also allows Genzyme to submit sections of an NDA on a rolling basis.

A Phase 2a trial of GZ/SAR402671 has begun recruiting patients with plans to enroll 9 treatment-naïve male adult patients with Fabry disease. There are approximately 10,000 patients with Fabry disease in the world.

“Becoming a Fast Track Program is an important milestone and we appreciate this designation from FDA,” said Genzyme’s Acting Head of Rare Diseases, Richard Peters, M.D, Ph.D. “We look forward to learning more about this small molecule, with the goal of providing more therapeutic options to the Fabry community as quickly as possible.”

The clinical trial will be conducted in numerous sites around the world, including 4 sites in the United States (Birmingham, Alabama; Atlanta, Georgia; Cincinnati, Ohio; and Fairfax, Virginia), Other sites are located in the Czech Republic, France, Poland, Russia, and England. For more information about the trial, visit clinicaltrials.gov/ct2/show/NCT02228460.


FDA Grants Fast Track Designation to Genzyme’s Investigational Substrate Reduction Therapy for the Treatment of Fabry Disease  [press release].  Cambridge, MA; Genzyme, a Sanafi company; April 28, 2015.  http://www.businesswire.com/news/home/20150427006341/en/FDA-Grants-Fast-Track-Designation-Genzyme%E2%80%99s-Investigational#.VT9Ttmb1jvI

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