FDA to speed up review of Fabry Therapy, says Genzyme
Genzyme, a Sanofi company, said on Tuesday that the United States Food and Drug Administration (FDA) has agreed to speed up review for the development of GZ/SAR402671. Sanofi’s GZ/SAR402671 is a new investigational oral substrate reduction therapy, which can treat Fabry disease.
Fabry disease is an inherited, potentially fatal disorder, which is characterized by the buildup of a particular type of fat. That fat can results in cell damage, which could lead to pain, kidney failure, hearing loss, stroke and heart attack.
Fast track Drug program of FDA has been designed to assist regular interactions with the review team of the agency to speed up to review a New Drug Application (NDA) for medicines with the ability to treat serious diseases. The program also offers an opportunity to submit sections of an NDA on a rolling basis before a company provides the complete application.
Currently, Genzyme has been enrolling participants in the Phase 2a trial of GZ/SAR402671. The company has planned to include nine treatment-naïve male adult patients with Fabry disease in the study. According to the company, the study is an international and multicenter study.
Genzyme’s GZ/SAR402671 is a glucosylceramide synthase inhibitor which can block the formation of glucosylceramide (GL-1), a key intermediate in the synthesis of GL-3. Richard Peters, Genzyme’s Acting Head of Rare Diseases, said, “Becoming a Fast Track Program is an important milestone and we appreciate this designation. 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”.
Genzyme, which is a Sanofi company, has pioneered the development of transformative therapies for individuals affected with rare and debilitating diseases for more than three decades.