FDA Approves New Treatment for Fabry Disease


Migalastat (Galafold, Amicus Therapeutics) is the first oral medication for the treatment of adults with Fabry disease, a rare genetic disorder.

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

Fabry disease is a rare genetic disorder caused by mutations in the alpha-galactosidase A gene located on the X-chromosome, according to the FDA. It results from a buildup of fat called globotriaosylceramide (GL-3) in blood vessels, the kidneys, the heart, the nerves, and other organs. Patients with Fabry disease can develop slowly progressive kidney disease, cardiac hypertrophy, arrhythmias, stroke, and early death.

The approval is based on efficacy data from a 6-month clinical trial involving 45 adults with Fabry disease. According to the findings, patients treated with migalastat over 6 months experienced a greater reduction in GL-3 in the blood vessels of the kidneys compared with patients treated with a placebo. The safety of migalastat was also studied in 4 clinical trials, which included a total of 139 patients with the disease.

The most commonly reported adverse effects associated with migalastat in the clinical trials were headache, nasal and throat irritation, urinary tract infection, nausea, and fever.

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

The treatment was approved under the FDA’s Accelerated Approval pathway. A further study is required to verify and describe the clinical benefits of migalastat, according to the press release.


FDA approves new treatment for a rare genetic disorder, Fabry disease [news release]. FDA’s website. https://bit.ly/2P1SdjN. Accessed August 13, 2018.

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