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.

RELATED: Metabolic Disorders Resource Center

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.

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