Rare Disease Report

Migalastat NDA Submitted to FDA for Treatment of Fabry Disease

DECEMBER 14, 2017
Mathew Shanley
Amicus Therapeutics announced this morning that it has submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of the potential Fabry disease treatment migalastat HCI.

Migalastat, an oral precision medicine, is intended to treat patients 16 and older with Fabry disease who have amenable mutations, which is approximately 35% to 50% of the patient population. In July, Amicus and the FDA met and agreed that sufficient data evaluating the safety and efficacy of the drug were available.

Fabry disease is a genetic lysosomal disorder caused by the deficiency of the enzyme alpha-galactosidase A (a-Gal A) that causes the degradation of lipids like globotriaosylceramide (GL-3). The build-up of fat can cause progressive damage to multiple organs, including the skin, eyes, kidneys, heart, and brain. Approximately 1 in every 40,000 to 60,000 males are effected by Fabry disease, per the NIH.

“Today marks the very first Amicus submission of a new drug application to the U.S. FDA,” said John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. “This important milestone is the culmination of a strong collaboration and commitment among the patients, physicians and Amicus employees who have spent more than a decade to advance migalastat. On the heels of our initial launch success for migalastat in Europe, our goal is to further expand access for more Fabry patients with amenable mutations in the U.S., Japan and other global geographies.  We look forward to working with the FDA during the review process and to a potential U.S. approval of migalastat in 2018.”

The drug, a chaperone therapy, previously received both Orphan Drug Designation and Fast Track designation from the FDA, and will now wait through a 60-day filing review period while the regulatory agency determines whether the NDA is complete and acceptable. It works by stabilizing alpha-Gal A so that the accumulation of disease substrate can be cleared in patients with these mutations.

Amicus’ road to its NDA submission for migalastat was not without challenges. In 2012, data from a clinical trial testing the efficacy of migalastat in Fabry patients did not meets its primary endpoint and few were optimistic that the drug would ever be approved. In 2015, Amicus announced another setback when it stated that the FDA wanted the company an additional study assessing the effects of migalastat on gastrointestinal symptoms to be conducted, a request that has since been withdrawn.

Migalastat has already been granted full approval as a first line therapy for long-term treatment of the same patient population by the European Commission (EC), and it is being marketed under the trade name Galafold. Amicus’ drug has also previously received approval in Switzerland, Israel, Australia and Canada, with regulatory submissions under review in Japan among other geographies.

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