Posted by Michael Wonder on 15 Dec 2017
Amicus Therapeutics submits new drug application to U.S. FDA for migalastat for treatment of Fabry Disease
14 December 2017 - Amicus Therapeutics submitted a new drug application to the U.S. FDA to request approval of the oral precision medicine migalastat hydrochloride ("migalastat") for the treatment of patients 16 years and older with Fabry disease who have amenable mutations.
The submission is based on existing clinical data, including reduction in disease-causing substrate (GL-3), as well as the totality of data from two Phase 3 pivotal studies in treatment-naïve (Study 011, or FACETS) and enzyme replacement therapy (ERT) switch patients (Study 012, or ATTRACT), as well as other completed clinical studies.
Migalastat previously received both orphan drug designation and fast track designation from the U.S. FDA. The FDA has a 60-day filing review period to determine whether the NDA is complete and acceptable for filing, after which the Agency will notify the Company. Amicus plans to communicate following written receipt of the Agency's decision.
