The Food and Drug Administration (FDA) has approved Galafold (migalastat; Amicus Therapeutics) for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data. The indication was approved under an accelerated approval pathway based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate. 

Fabry disease is a rare, progressive genetic disorder characterized by a GLA mutation located on the X-chromosome. Galafold, a pharmacological chaperone of alpha-galactosidase A (alpha-Gal A), is the first oral precision medication approved for Fabry disease. “Thus far, treatment of Fabry disease has involved replacing the missing enzyme that causes the particular type of fat buildup in this disease,” said Julie Beitz, MD, director of the Office of Drug Evaluation III in FDA’s Center for Drug Evaluation and Research. “Galafold differs from enzyme replacement in that it increases the activity of the body’s deficient enzyme.” 

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The approval of Galafold was based on a 6-month, placebo-controlled trial involving 45 patients with Fabry disease (all had amenable GLA variants). The major efficacy outcome measure was the average number of GL-3 inclusions per KIC in renal biopsy samples before and after treatment. Results showed that 52% of Galafold-treated patients had a ≥50% reduction from baseline in the average number of GL-3 inclusions per KIC (median change from baseline: -0.04) vs 45% of patients in the placebo group (median change from baseline: -0.03). Headache, nasopharyngitis, urinary tract infection, nausea, and pyrexia were the most common adverse effects associated with Galafold. 

Galafold will be available as 123mg capsules in blister packs containing 14 capsules. The product will be shipped to a limited distribution network immediately. 

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