Rare Daily Staff
The U.S. Food and Drug Administration has accepted the New Drug Application and granted priority review status for Amicus Therapeutics migalastat, an experimental therapeutic for patients 16 years and older with certain forms of Fabry disease, a progressive rare genetic disease that damages organs throughout the body.
Priority review status accelerates the review time for a drug seeking marketing approval from 10 months to a goal of six months from the day of acceptance of filing. It is conferred on drugs that may offer major advances in treatment or may provide a treatment where no adequate therapy exists. Migalastat also has Orphan Drug Designation and Fast Track designation from the FDA.
Migalastat is a first-in-class chaperone therapy approved in the European Union as a monotherapy for Fabry disease in patients with amenable mutations. Migalastat works by stabilizing the body’s own dysfunctional enzyme, so it can clear the accumulation of disease substrate in patients who have amenable mutations.
A proprietary in vitro assay has been used to classify more than 1,000 known mutations to the GLA gene, the faulty gene that causes Fabry disease. Amicus said there are 348 GLA mutations that have been determined to be amenable to migalastat, which represent between 35 percent and 50 percent of the currently diagnosed Fabry population.
Migalastat is approved in Switzerland, Israel, Australia, South Korea, and Canada, with regulatory submissions under review in the United States, Japan, and Taiwan.
February 12, 2018