Amicus May Not Submit NDA For Migalastat Monotherapy In US By Year End

(RTTNews) - Amicus Therapeutics (FOLD), a biotechnology company focused on rare and orphan diseases, on Friday announced additional regulatory guidance from the U.S. Food and Drug Administration on the oral small molecule pharmacological chaperone migalastat for the treatment of Fabry disease.

Amicus said it 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 FDA, 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.

Also, the FDA 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.