Migalastat(Amicus/GSK) disappoints in Phase III trial for Fabry Disease

Amicus Therapeutics and GSK announced the 6-month primary treatment period results from the first Phase III global registration study (Study 011) 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.

GL-3 clearance from the kidney interstitial capillaries is used as a marker of treatment effect in Fabry Disease. Capillary GL-3 after 6 months of treatment with migalastat HCl was compared to placebo. Patients with evaluable baseline biopsies, 13/32 (41%) in the migalastat HCl treatment group demonstrated a 50% or greater reduction in kidney interstitial capillary GL-3 after 6 months of study treatment versus 9/32 (28%) in the placebo group. This difference did not achieve statistical significance. A second Phase III global registration study (Study 012) is also underway to compare open-label migalastat HCl to enzyme replacement therapy.