Amicus Therapeutics holds pre-NDA meeting for Fabry disease drug

Amicus Therapeutics on Tuesday announced that a pre-New Drug Application (NDA) meeting was held with the U.S. Food and Drug Administration (FDA) to discuss the oral, small-molecule pharmacological chaperone migalastat for the treatment of Fabry disease.

"Our collaborative pre-NDA meeting represents a significant milestone for the Fabry community in the United States and is a great example of FDA and industry working together to advance innovative therapies for people living with debilitating genetic disorders," Amicus Therapeutics CEO and Chairman John Crowley said.

Fabry disease is an inherited lysosomal storage disorder caused by deficiency of an enzyme called alpha-galactosidase. Symptoms include pain, kidney failure and increased risk of heart attack and stroke. The mechanism of action of migalastat represents a new personalized medicine option for Fabry patients who have amenable mutations.

"The guidance provided by FDA during the pre-NDA meeting further reinforces our confidence in the NDA package and post-marketing confirmatory study we are preparing for submission by the end of this year,” Crowley said. “In addition, our marketing submission for migalastat in Europe is already being reviewed under accelerated assessment and an opinion is expected by year-end. With defined regulatory pathways for migalastat in both the U.S. and E.U., we are rapidly executing our global strategy to bring this novel personalized medicine to as many people living with Fabry disease as quickly as possible."

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