Amicus Says FDA Didn't Grant Accelerated Approval For Migalastat; Stock Plunges

(RTTNews) - Amicus Therapeutics (FOLD) announced its planned regulatory pathway to collect additional data to support full approval for the oral precision medicine migalastat for Fabry disease. The U.S. Food and Drug Administration indicated that kidney globotriaosylceramide (GL-3) is currently not considered a basis for an accelerated approval under Subpart H.

FOLD closed Monday's regular trading at $8.32, down $0.58 or 6.52 percent. In the after-hours, the stock dropped $2.22 or 26.68 percent.

Following several collaborative discussions with the U.S. Food and Drug Administration, including the receipt of final written minutes from an in-person Type B meeting, the Company plans to collect additional data on gastrointestinal (GI) symptoms in Fabry patients who have an amenable mutation.

Key elements of the additional data generation required for full approval of migalastat in the United States may include: Randomized, 12-month, placebo-controlled pivotal "cross-over" study in treatment naïve Fabry patients who have an amenable mutation and GI symptoms; Crossover study design provides sufficient powering with a small number of patients (~35 patients planned); Primary endpoint to assess diarrhea based upon established FDA irritable bowel syndrome (IBS) guidance.

Amicus said it is working with FDA to finalize the clinical protocol and plans to initiate enrollment in 2017, with data expected in 2019.Upon successful NDA filing, the review will be based upon these new data and the totality of the data from all prior migalastat studies; Intermediate Expanded Access Program (EAP) planned to ensure short-term access to migalastat for U.S. patients who are currently on ERT and meet EAP requirements; U.S. patients currently enrolled in ongoing clinical extension studies will continue to receive migalastat.