The primary analysis compared the number of responders in the migalastat HCl versus placebo groups, based on a 50% or greater reduction in interstitial capillary globotriaosylceramide (GL-3) during the 6-month, double-blind treatment period. GL-3 – also referred to as peritubular capillary (PTC) inclusions – is measured in kidney biopsies. Pathologists blinded to biopsy sequence used the published, quantitative Barisoni Lipid Inclusion Scoring System with virtual microscopy (BLISS-VM) for the histological evaluation of interstitial capillary GL-3 in Study 011. BLISS is a more sensitive scoring system to measure GL-3 inclusions in PTCs compared to the semi-quantitative methodology used in previous pivotal studies of enzyme replacement therapy (ERT) for Fabry disease. Secondary endpoints for Study 011 include safety and tolerability, urine GL-3 and kidney function.About Migalastat HCl
Amicus Therapeutics And GlaxoSmithKline Announce Top Line 6-Month Primary Treatment Period Results From First Phase 3 Fabry Monotherapy Study
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