FDA grants accelerated approval for Galafold treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data.--Amicus Therapeutics.

Amicus Therapeutics has announced that the FDA has granted accelerated approval of Galafold (migalastat) 123 mg capsules. Galafold is an oral, precision medicine for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data.

Galafold was approved under the Subpart H Accelerated Approval pathway based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate. As a condition of accelerated approval, Amicus Therapeutics will continue to study Galafold in a confirmatory Phase IV program. Galafold is a capsule taken once every other day, at the same time of day. The FDA has approved Galafold for 348 amenable GLA variants. Galafold is the first oral medicine for Fabry disease, and the first new therapy approved to treat Fabry disease in the United States in more than 15 years.

“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, M.D., director of the Office of Drug Evaluation III in FDA’s Center for Drug Evaluation and Research..

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.The safety of Galafold was studied in four clinical trials which included a total of 139 patients with Fabry disease.