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FDA Approves Galafold for Fabry Disease in Adults

First Oral Drug Increases Activity of the Body’s Deficient Enzyme

The FDA has approved Galafold (migalastat, Amicus Therapeutics), the first oral medication for the treatment of adults with Fabry disease. The drug is indicated for adults who have a genetic mutation determined to be responsive to treatment with Galafold based on laboratory data.

“Thus far, treatment of Fabry disease has involved replacing the missing enzyme that causes the particular type of fat buildup in this disease. Galafold differs from enzyme replacement in that it increases the activity of the body’s deficient enzyme,” said Julie Beitz, MD, director of the Office of Drug Evaluation III in the FDA’s Center for Drug Evaluation and Research.

Fabry disease is a rare, inherited disorder caused by mutations in the alpha-galactosidase A (GLA) gene located on the X-chromosome; it affects both males and females and results from the buildup of globotriaosylceramide (GL-3) in blood vessels, nerves, the kidneys, the heart, and other organs. It is estimated that classic Fabry disease (the most severe type) affects approximately one in 40,000 males. The later-onset type is more frequent, and in some populations, may occur in one in 1,500 to 4,000 males. Patients with Fabry disease develop slowly progressive kidney disease, cardiac hypertrophy, arrhythmias, stroke, and early death.

The efficacy of Galafold was demonstrated in a six-month, placebo-controlled clinical trial in 45 adults with Fabry disease. In this trial, patients treated with Galafold over six months had a greater reduction in globotriaosylceramide (GL-3) in blood vessels of the kidneys (as measured in kidney biopsy samples) as compared to patients taking placebo. The safety of Galafold was studied in four clinical trials that included a total of 139 patients with Fabry disease. The most common adverse drug reactions in patients taking Galafold in the trials were headache, nasopharyngitis, urinary tract infection, nausea, and pyrexia.

A further study is required to verify and describe the clinical benefits of Galafold, and the manufacturer will be conducting a confirmatory clinical trial of Galafold in adults with Fabry disease. Galafold was granted priority review and orphan drug designations.

Source:, August 10, 2018

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