Persistence Pays Off For Amicus: Positive Migalastat Data In Hand

Amicus will return to FDA with positive data on its oral small molecule chaperone migalastat in Fabry patients after a Phase III trial showed the oral drug had comparable efficacy to existing enzyme replacement therapies.

Aug. 20 was a day of vindication for Amicus Therapeutics Inc., which now has positive Phase III results on its oral Fabry disease therapy migalastat and a comprehensive data package to submit to FDA – two years after the first portion of a Phase III trial disappointed and a year after the rare disease drug developer lost its big pharma partner.

Migalastat could become the first oral small molecule treatment for a portion of patients with the rare inherited disease, who are currently treated with enzyme replacement therapies that require infusions...

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