Sanofi's (SNY +0.4%) Genzyme unit initiates a Phase 2a clinical trial evaluating the pharmacodynamics of GZ/SAR402671, a new once-daily oral therapy for Fabry disease, a rare lysosomal storage disorder that results in the buildup of a type of fat, globotriasylceramide (GL-3), in the body's cells caused by a deficiency in a certain enzyme. It affects ~one in 50K males, predominantly, or ~10K total people worldwide.
The primary endpoint of the nine-patient 26-week study is the reduction in accumulated GL-3 from the skin capillary endothelium. After its completion, all patients will have the option to enroll in an extension study.
Genzyme's current offering for Fabry, Fabrazyme (agalsidase beta), replaces the deficient enzyme, alpha-galactosidase A. The FDA approved the intravenously-administered therapy in April 2003. It generated over $500M in sales for Sanofi last year. Annual therapy costs ~$200K per patient.
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