FDA Approves Migalastat for Fabry Disease

The US Food Drug Administration (FDA) has approved migalastat (Galafold), the first oral medication indicated for adult patients with Fabry disease.

The drug, indicated for adult patients who have a genetic mutation determined to be responsive to the therapy, was approved on the backing of a six-month, placebo-controlled clinical trial involving 45 patients with Fabry disease. Patients treated with the therapy reported greater reductions in globotriaoslyceramide (GL-3) in blood vessels of the kidney versus patients administered placebo.

Migalastat’s safety was measured in 4 clinical trials involved 139 patients with Fabry disease. The most common adverse events patients were headache, nasal and throat irritation, urinary tract infection, nausea, and fever.

Fabry disease is a rate, inherited disease cause by mutations in the alpha-galactosidase A (GLA) gene located on the X-chromosome. The classic form of the disease—incidentally, its most severe type—affects approximately 1 in 40,000 males, while the later-onset form of the disease may occur in 1 in 1500-4000 males.

The disease could lead to progressive kidney disease, enlargement of the heart, arrhythmias, stroke, and early death. Julie Beitz, MD, director of the Office of Drug Evaluation III in FDA’s Center for Drug Evaluation and Research, said migalastat has been proven to attack the condition with an effective, novel approach.

“Thus far, treatment of Fabry disease has involved replacing the missing enzyme that causes the particular type of fat buildup in this disease,” Beitz said in a statement. “Galafold differs from enzyme replacement in that it increases the activity of the body’s deficient enzyme.”

Migalastat, to be marketed by Amicus Therapeutics, was approved under the Accelerated Approval pathway, designated for FDA-considered drugs for serious conditions that have an unmet medical need. It was also granted Priority Review designation, and Orphan Drug designation.