Rare Disease Report

FDA Approves First Oral Medication for Fabry Disease

AUGUST 10, 2018
Kristi Rosa
The US Food and Drug Administration (FDA) has approved Amicus Therapeutics' migalastat (Galafold), which is the first oral medication for the treatment of adults with Fabry disease, a rare and serious genetic disease. The drug is indicated specifically for adults with the disease who have a genetic mutation determined to be amenable to treatment with migalastat 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,” Julie Beitz, MD, director of the Office of Drug Evaluation III in the FDA’s Center for Drug Evaluation and Research, said in a recent statement. “Galafold differs from enzyme replacement in that it increases the activity of the body’s deficient enzyme.”

Efficacy of the treatment was shown in a placebo-controlled clinical trial which enrolled 45 adults with Fabry disease. For the trial, patients who were administered migalastat for the duration of 6 months were found to have a larger reduction in globotriaosylceramide (GL-3) in blood vessels of the kidneys compared with those who received the placebo. The drug’s safety was evaluated in 4 clinical trials which included 139 patients with Fabry disease in total.

A headache, nasopharyngitis, urinary tract infection, nausea, and pyrexia were listed as the most common adverse drug reactions observed in patients taking the medication.

Migalastat was approved using the Accelerated Approval pathway, according to the FDA. This pathway allows for the approval of drugs for serious conditions where there is an unmet medical need and where a drug has demonstrated likeliness to provide clinical benefit to patients.

Further study is required to verify and describe migalastat’s clinical benefit, according to the FDA, and the sponsor will conduct a confirmatory clinical trial evaluating the drug in adults with Fabry disease.

Previous to this approval, migalastat was granted Priority Review designation.
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Fabry disease, although rare, is estimated to affect about 1 in 40,000 males. The later-onset type of the disease is more common, and as such, may occur in 1500 to 4000 males in some populations. Those with Fabry disease are known to develop progressive kidney disease, cardiac hypertrophy, arrhythmias, stroke, and early death.

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