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news.Sanofi's Genzyme has secured fast track designation from the US Food and Drug Administration (FDA) for the development of GZ/SAR402671, a new investigational oral substrate reduction therapy to treat Fabry disease, a rare lysosomal storage disorder.
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Fabry disease results in abnormal tissue deposits of a particular fatty substance (called globotriaosylceramide, also referred to as GL-3 or Gb3) throughout the body. It is characterized by excessive accumulation of the lipid GL-3 in various organs and tissues.
Currently, the company is planning to enroll nine treatment-naïve male adult patients with Fabry disease in this international, multicenter Phase IIa trial of GZ/SAR402671, a glucosylceramide (GL-1) synthase inhibitor that blocks the formation of GL-1, a key intermediate in the synthesis of GL-3.
Genzyme Rare Diseases acting head Richard Peters said: "Becoming a Fast Track Program is an important milestone and we appreciate this designation from FDA.
"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."