Amicus Therapeutics Inc. said the U.S. Food and Drug Administration granted accelerated approval to its medicine Galafold to treat Fabry disease, a rare genetic disorder.
The approval covers 123-milligram Galafold, or migalastat, capsules for adult patients with Fabry disease who have a mutation in the galactosidase alpha, or GLA, gene determined to be responsive to treatment.
Fabry disease is an inherited disorder that results from the buildup of a particular type of fat called globotriaosylceramide, or GL-3, in the blood vessels, the kidneys, the heart, nerves and other organs. Patients with Fabry disease develop slowly progressive kidney disease, enlargement of the heart, abnormal heart rhythm, stroke and early death.
Amicus noted that Galafold is the first oral medicine for Fabry disease and the first new therapy approved to treat the condition in the U.S. in more than 15 years.
In the U.S., it is estimated that more than 3,000 people are living with Fabry disease, and more than an estimated 50% of these diagnosed patients are currently untreated. Globally, it is estimated that 35% to 50% of patients with Fabry disease may have an amenable GLA variant, Amicus said in an Aug. 10 news release.
Galafold was approved using the accelerated approval pathway, under which the FDA may approve drugs for serious conditions where there is an unmet medical need and where a drug is shown to have certain effects that are reasonably likely to predict a clinical benefit to patients.
Amicus said that as a condition of the accelerated approval, it will continue to study Galafold in a confirmatory phase 4 program.
The Cranbury, N.J.-based biotechnology company said the approval was based on positive data from a phase 3 trial, consisting of 45 patients, which showed the effectiveness of the medicine, including a reduction in the amount of damaging disease substrate accumulated in the kidney capillaries.
Amicus will launch Galafold immediately and will begin shipping to a limited distribution network in the coming week.
Galafold, which was granted priority review status by the FDA in February, is already approved in the EU, Japan, Australia, Canada, Israel, South Korea and Switzerland. Its approval is pending in Taiwan.