Amicus Therapeutics Files for FDA Approval of Migalastat to Treat Fabry Disease
Amicus Therapeutics recently submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for its investigational oral medicine migalastat for treatment of patients 16 years and older with Fabry disease who have so-called “amenable” mutations. The NDA seeks migalastat’s approval in the U.S. as an alternative to intravenous enzyme…