FDA panel reviews Fabrazyme

FDA's Endocrinologic and Metabolic Drugs Advisory Committee voted 14-1 on Monday that clearance of globotriasylceramide ( GL-3) from endothelial kidney cells, the surrogate marker in Genzyme's pivotal trial of its Fabrazyme agalsidase beta, is reasonably likely to predict clinical benefit in Fabry's disease patients. The panel also said that the company demonstrated that Fabrazyme had a significant effect on GL-3 clearance from various cell types, including endothelial kidney cells, in Fabry's disease patients.

Most committee members said that the benefits of Fabrazyme outweigh the most serious risks identified in trials of Fabrazyme, infusion site reactions. There also was a consensus that data obtained to date do not indicate that antibody formation decreases the efficacy of Fabrazyme and that the effects of antibodies should be studied after the product is approved. ...