BioCentury
ARTICLE | Clinical News

Amicus submits NDA for Fabry candidate

December 15, 2017 7:47 PM UTC

Amicus Therapeutics Inc. (NASDAQ:FOLD) submitted an NDA to FDA for migalastat (AT1001) to treat patients 16 years and older with Fabry disease who have amenable mutations.

In July, Amicus said FDA would no longer require an additional Phase III study of the candidate to support an NDA submission, reversing the agency's November 2016 opinion that reductions in globotriaosylceramide (GL-3), a biomarker used in the company’s Phase III FACETS trial, could not serve as the basis for accelerated approval (see BioCentury, July 14)...

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