2:06 PM
Feb 12, 2018
 |  BC Extra  |  Company News

Priority Review for Amicus' migalastat

Editor's Note: This article was updated on Feb 12, 2018 at 4:44 PM PST

FDA accepted and granted Priority Review to an NDA from Amicus Therapeutics Inc. (NASDAQ:FOLD) for migalastat (AT1001) to treat Fabry's disease in patients 16 years and older who have amenable mutations. The PDUFA date is Aug. 13.

The NDA is based on data from the Phase III FACETS and ATTRACT trials.

In July, Amicus said FDA would no longer require an additional Phase III study to support an NDA for migalastat after the company presented new cardiac and renal data, new analyses of existing data previously reviewed by European regulators, long-term data from extension studies, patient experiences with the drug and lack of treatment options in the U.S. In November 2016, Amicus said FDA had determined that reductions in globotriaosylceramide (GL-3), a biomarker used in the FACETS trial, could not serve as the basis for accelerated approval. To fulfill FDA’s requirements, Amicus would have had to conduct a third Phase III trial in about 35 Fabry’s disease patients with amenable mutations (see BioCentury Extra, July 11, 2017).

Migalastat has Orphan Drug and Fast Track designations from FDA.

The small molecule stabilizer of alpha galactosidase A is approved as Galafold in the EU for the indication and is under review in Japan.

Amicus added $0.09 to $14.81 on Monday.

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