3.235.65.220
dgid:
enl:
npi:0
Journal Scan
Metabolic

Migalastat Improves Diarrhea in Some Patients With Fabry Disease

Posted on

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.

Read more here.

Reference

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.
doi: 10.1186/s13023-018-0813-7.

-Advertisement-
Related Articles
Society for InherIted Metabolic Disorders Provides Update on Annual Meeting Amidst COVID-19 Outbreak
Jun 02, 2020
FDA Approves New Treatment for Adults with Cushing’s Disease
Jun 01, 2020
FDA Grants Rare Pediatric Disease Designation for Barth Syndrome Treatment
May 31, 2020
-Advertisement-
-Advertisement-
-Advertisement-
-Advertisement-
-Advertisement-