Genzyme starts mid-stage trial of oral Fabry disease therapy
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.
Hoe toevallig...onze oude bekende Genzyme. Genzyme zit wat betreft de product leads van het door Pharming overgenomen TRM ook al in het vaarwater met de Ziekte van Pompe. Ook hier moet Pharming opschieten dus.
Alhoewel.. ook Genzyme moet opschieten want Amicus is nog verder met de Ziekte van Fabry:
"Amicus is planning to file for an NDA in 2015 for its Fabry drug Migalastat."
"Amicus (NASDAQ:FOLD) has just been added to the Nasdaq Biotechnology Index and has shown intent to file for the marketing approval of its Fabry treatment with the EMA and FDA. The shares have already rallied form below $2 to above $8 and have the capability to go even higher on important catalysts coming up in 2015."