Posted by Michael Wonder on 12 Feb 2018
U.S. FDA files new drug application under priority review for migalastat for treatment of Fabry disease
12 February 2018 - Six month PDUFA goal date is 13 August 2018.
Amicus Therapeutics today announced that the U.S. FDA has accepted the new drug application for filing under priority review for the oral precision medicine migalastat hydrochloride for the treatment of patients 16 years and older with Fabry disease who have amenable mutations. The Prescription Drug User Fee Act goal date for the FDA decision is 13 August 2018.
Migalastat previously received both orphan drug designation and fast track designation from the U.S. FDA. The FDA’s priority review status accelerates the review time from 10 months to a goal of six months from the day of acceptance of filing and is given to drugs that may offer major advances in treatment or may provide a treatment where no adequate therapy exists.
The NDA submission for migalastat is based on clinical data from completed studies, including reduction in disease-causing substrate, as well as the totality of data from two Phase 3 pivotal studies in treatment-naïve (Study 011, or FACETS) and enzyme replacement therapy switch patients (Study 012, or ATTRACT).
