Amicus Theraputics presents additional data from Fabry monotherapy study Amicus Therapeutics announced additional 6-month results from the first ongoing Phase 3 global registration study of investigational oral migalastat HCl monotherapy in males and females with Fabry disease who had genetic mutations identified as amenable to migalastat HCl in a cell-based assay. Study 011 consists of a 6-month, double-blind period when subjects received migalastat HCl 150 mg or placebo, a 6-month open label-follow up period when all patients received migalastat HCl, and an ongoing 12-month open-label extension. Initial top-line Stage 1 results previously reported in December, 2012 for the primary endpoint in Stage 1 did not meet statistical significance. The pre-specified primary and secondary analyses of the primary endpoint numerically favored migalastat HCl over placebo. In the primary responder analysis, 13/32 in the migalastat HCl group versus 9/32 in the placebo group demonstrated a 50% or greater reduction in kidney interstitial capillary GL-3 from baseline to month 6. Taken alone the secondary analysis of the absolute percent change in kidney interstitial capillary GL-3 from baseline to month 6 showed a median reduction of 41% in the migalastat HCl group versus a median reduction of 6% in the placebo group. Stage 1 results were highlighted in an oral platform presentation at the Lysosomal Disease Network WORLD Symposium.
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