- Medical Updates

The Food and Drug Administration ( FDA ) has granted Fast Track designation for the development of GZ/SAR402671, a new investigational oral substrate reduction therapy for the treatment of Fabry disease.

FDA’s Fast Track Drug Development Program is designed to facilitate frequent interactions with the FDA review team to expedite the clinical development and review of a New Drug Application ( NDA ) for medicines with the potential to treat serious or life-threatening conditions and address unmet medical needs for such disease or conditions. It also provides the opportunity to submit sections of an NDA on a rolling basis before a sponsor submits the complete application.

Genzyme is currently enrolling patients in its phase 2a trial of GZ/SAR402671, and plans to enroll nine treatment-naïve male adult patients with Fabry disease in this international, multicenter study.

Fabry disease is a rare lysosomal storage disorder that results in abnormal tissue deposits of a particular fatty substance ( called globotriaosylceramide, also referred to as GL-3 or Gb3 ) throughout the body.
GZ/SAR402671 is a glucosylceramide synthase inhibitor that blocks the formation of glucosylceramide ( GL-1 ), a key intermediate in the synthesis of GL-3.

Fabry disease is an inherited and progressive condition that is characterized by excessive accumulation of the lipid GL-3 in various organs and tissues.
Early symptoms include significant pain, gastrointestinal disturbances, as well as other manifestations, and over time, patients may experience life threatening renal, cardiac and cerebrovascular events.
As a result, patients with Fabry disease typically have a shortened life span.
Fabry disease affects both males and females, with approximately 10,000 diagnosed patients in the world. ( Xagena )

Source: Genzyme, 2015