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Amicus Therapeutics seeks US approval for Fabry disease drug

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Amicus Therapeutics seeks US approval for Fabry disease drug

Amicus Therapeutics Inc. has submitted a new drug application to the U.S. Food and Drug Administration for migalastat to treat patients 16 years and older with Fabry disease who have amenable mutations.

Fabry disease is an inherited disorder that results from the buildup of a particular type of fat in the body's cells.

Migalastat has previously been awarded fast track and orphan drug designations by the FDA.

The drug is already approved in the EU under brand name Galafold as a first line therapy for long-term treatment of patients with a confirmed diagnosis of Fabry disease.