Nearly half of Fabry patients have amenable or responsive mutations to Galafold (migalastat) but show different responses to the therapy, according to a Swiss population study. The study found that patients with higher residual activity of the alpha-galactosidase A (GLA) enzyme — which is deficient in Fabry patients —…

Amicus Therapeutics‘ Galafold (migalastat) provides clinical benefit to patients carrying amenable Fabry disease mutations, regardless of disease severity, a Phase 3 trial shows. The findings of the study, “Efficacy of the pharmacologic chaperone migalastat in a subset of male patients with the classic phenotype of Fabry…

Amicus Therapeutics announced the beginning of the availability of Galafold (migalastat) to treat Fabry disease in Japan, making this the first oral precision medicine available for patients with an amenable mutation in that country. Fabry disease is caused by mutations in the GLA gene, which lead to a defective…

Galafold (migalastat) significantly reduces diarrhea in patients with Fabry disease and “amenable” mutations — that can respond to Galafold treatment — according to a recent analysis of the FACETS study’s results. This result supports previous evidence from the same trial showing that Galafold improves gastrointestinal symptoms, including diarrhea,…

Galafold (migalastat) capsules of 123 mg were approved in Japan to treat Fabry disease patients, ages 16 and older, with mutations amenable to the treatment, Amicus Therapeutics recently announced. This move by Japan’s Ministry of Health, Labour and Welfare makes Galafold, developed by Amicus, the first and only oral precision medicine…

Amicus Therapeutics recently submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for its investigational oral medicine migalastat for treatment of patients 16 years and older with Fabry disease who have so-called “amenable” mutations. The NDA seeks migalastat’s approval in the U.S. as an alternative to intravenous enzyme…