Amicus Therapeutics on Friday said that the FDA granted accelerated approval to Galafold (migalastat) for the treatment of adults with a confirmed Fabry disease diagnosis and an amenable galactosidase alpha gene variant. The drugmaker indicated that it will launch the therapy, which is the first oral medication for the treatment of adults with Fabry disease, immediately and begin shipment to a limited distribution network in the next few days. Amicus CEO John Crowley remarked "this FDA approval of Galafold is a transformative moment for people in the US living with Fabry disease, as it gives adult patients with amenable GLA variants a new treatment option for the first time in more than 15 years."
According to Amicus, approval of the drug was backed by data from Study 011, with study results illustrating that Galafold significantly reduced kidney interstitial capillary globotriaosylceramide levels versus placebo.
The FDA agreed to review a new drug application of Galafold from Amicus in July last year, after having previously rebuffed the drugmaker's request seeking accelerated approval of the drug, while requesting additional studies. The therapy has been granted fast track designation and priority review by the agency.
For related analysis, see ViewPoints: Having been ready for years, Amicus finally sees US approval for Galafold.
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