Rare Lysosomal Disorder Therapy Designated Fast Track Status
Genzyme announced that the Food and Drug Administration (FDA) has granted Fast Track designation for the development of GZ/SAR402671, an oral substrate reduction therapy for the treatment of Fabry disease.
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 a rare lysosomal storage disorder that results in abnormal tissue deposits of a particular fatty substance called globotriaosylceramide throughout the body.
Genzyme is currently enrolling patients in the Phase 2a trial of GZ/SAR402671. The company plans to enroll nine treatment-naïve male adult patients with Fabry disease in this international, multicenter study.
For more information call (800) 745-4447 or visit Genzyme.com.