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FDA gives Amicus OK for Galafold for Fabry disease

The Food and Drug Administration has cleared Amicus Therapeutics U.S.'s Galafold (migalastat), the first oral medication for the treatment of adults with Fabry disease, the agency announced recently.

Galaford is indicated for adults with Fabry disease who have a genetic mutation determined to be responsive to treatment with Galafold based on laboratory data.

Fabry disease is a rare and serious genetic disease that results from buildup of a type of fat called globotriaosylceramide in blood vessels, the kidneys, the heart, the nerves and other organs.

"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," director of the Office of Drug Evaluation III in FDA's Center for Drug Evaluation and Research Julie Beitz said, in a press statement.

Fabry disease, which affects men and women, is an inherited disorder caused by mutations in the alpha-galactosidase A (GLA) gene located on the X-chromosome. It is estimated that classic Fabry disease — the most severe type — affects approximately one in 40,000 males, according to the FDA.

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 (enlargement of the heart), arrhythmias (abnormal heart rhythm), 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 on placebo.

Galafold was granted Priority Review designation and Orphan Drug designation. The latter designation provides incentives to assist and encourage the development of drugs for rare diseases.
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