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Amicus' Oral Fabry Drug Priced To Compete Against Traditional ERT

 
• By Jessica Merrill

FDA approved Galafold under accelerated approval, paving the way for the launch of the first oral therapy to treat a subset of patients with the rare condition Fabry disease. Amicus priced Galafold at $315,000 per year.

Bumpy road crossing plains at sunset

Amicus Therapeutics Inc.'s Galafold (migalastat) will launch in the US as the first oral therapy for a subset of patients with the rare condition Fabry disease, after a winding development road that extended back more than a decade.

The company announced FDA approval of Galafold Aug. 10 for patients with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on an in vitro test, about 35%-50% of the broader Fabry population

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