Migalastat Improves Diarrhea in Some Patients With Fabry Disease
A subset analysis from the phase 3 FACETS trial showed that the recently approved oral drug migalastat (Galafold) significantly reduced the incidence of diarrhea in patients with Fabry disease and a mutation targeted by migalastat. Gastrointestinal signs and symptoms affect almost half of Fabry patients and greatly compromise function and quality of life. After 6 months of treatment, 43% of patients in the migalastat arm had clinically meaningful improvement in diarrhea symptoms from baseline compared with 11% of patients in the placebo arm (P = .02). The difference between the migalastat and placebo arms was even greater when analyses were restricted to patients with diarrhea at baseline (71% vs 20%; P = .02). Diarrhea improvement correlated with a decrease in mean number of kidney peritubular capillary globotriaosylceramide (GL-3) inclusions. The study was funded by Amicus Therapeutics, Inc.
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Schiffmann R, Bichet DG, Jovanovic A, et al. Migalastat improves diarrhea in patients with Fabry disease: clinical-biomarker correlations from the phase 3 FACETS trial. Orphanet Journal of Rare Diseases. 2018;13:68.