Amicus Therapeutics (FOLD) and GlaxoSmithKline (GSK) announced the 6-month primary treatment period results from the first 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. The pre-designated primary endpoint was the number of patients who demonstrated a 50% or greater reduction in kidney interstitial capillary globotriaosylceramide-3 after 6 months of treatment with migalastat HCl compared to placebo. 13/32 (41%) in the migalastat HCl treatment group achieved the endpoint versus 9/32 (28%) in the placebo group. This difference did not achieve statistical significance. A pre-specified secondary analysis assessing 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. To date, no drug-related serious adverse events have been observed.
- Pharmaceuticals & Drug Trials