FDA Reverses Course, Will Allow Amicus to Submit Fabry Disease NDA

The FDA has accepted Amicus Therapeutics’ submission of its rare disease drug for review, a reversal from its decision under the Obama administration.

Last year, the agency told Amicus it would need to conduct further safety studies for migalastat, its drug candidate for Fabry disease. In December, the company agreed to conduct a further 12-month study on Fabry patients with gastrointestinal symptoms.

This week, however, Amicus announced the agency deemed its data sufficient to support an NDA submission, saying the additional Phase III data would not be necessary.