Amicus Therapeutics: Recent Developments, Financial Overview

In August, the FDA approved Amicus Therapeutics’ (FOLD) Galafold for the treatment of adults with a confirmed diagnosis of Fabry disease along with an amenable galactosidase alpha gene variant. The FDA’s approval of Galafold was according to the accelerated approval process based on the decrease in KIC GL-3 (kidney interstitial capillary cell globotriaosylceramide) substrate. Studies estimate that more than 3,000 individuals in the United States have Fabry disease.