Amicus Therapeutics Provides U.S. Regulatory Update for Migalastat Monotherapy

Amicus Therapeutics

, a biotechnology company at the forefront of therapies for rare and orphan diseases, today announced additional regulatory guidance from the U.S. Food and Drug Administration (FDA) on the oral small molecule pharmacological chaperone migalastat for the treatment of Fabry disease. Amicus has received final FDA minutes from the September pre-NDA meeting and has conducted additional follow-up interactions with the Agency this week. In conjunction with the Agency, Amicus is further evaluating several U.S. pathways including potentially generating additional data on migalastat's effect on gastrointestinal symptoms in Fabry disease to support submission requesting full approval as well as a Subpart H strategy. In addition, the Agency has requested further integration of existing clinical data across studies which will require more time to complete. Based on this guidance from the FDA, Amicus does not anticipate being in a position to submit the NDA for migalastat monotherapy in the United States by the end of this year. The timing of an NDA submission will be based on the determination of the optimal regulatory pathway. "Amicus remains committed to making migalastat available to Fabry patients with amenable mutations in the U.S. as rapidly as possible. We are appreciative of the FDA's ongoing collaboration in this program," stated John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics.