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Amicus Eyes Broad Future Label For Migalastat After Huge Filing Setback

 
• By Emily Hayes

US FDA recommends company start a new study of oral Fabry disease drug, to be designed using the agency's irritable bowel syndrome guidance for measuring efficacy in diarrhea.

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Amicus Therapeutics Inc.says it is pursuing a broad label for full approval of its oral migalastat in Fabry disease after announcing that FDA rejected its plan for accelerated approval and called for a new trial.

Migalastat is designed to selectively and reversibly bind with high affinity to the active sites of certain mutant forms of alpha-Gal A in Fabry disease, a lysosmal storage disorder. The drug was approved for Fabry disease with amenable mutations in May in Europe, where it is marketed as Galafold and is being rolled out gradually in various countries

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