Galafold (migalastat) is a medication developed by Amicus Therapeutics as a treatment for Fabry disease.

It was approved by the U.S. Food and Drug Administration in August 2018 under an accelerated approval program for medications that fill an unmet medical need and that have shown likely clinical benefits to patients but require further studies to verify their potential. The therapy was also granted priority review and orphan drug designation.

The European Medicines Agency (EMA) authorized Galafold for use in the European Union in 2016. It is also approved in Switzerland, Israel, Australia, South Korea, and Canada.

Fabry disease patients require lifelong treatments to manage the complications the condition causes in multiple organs. Enzyme replacement therapy with recombinant human alpha-gal A (agalsidase beta) or gene-activated alpha-gal A (agalsidase alfa) is the current standard of care for Fabry disease.

How Galafold works

Fabry disease is a genetic condition caused by mutations of the GLA gene. These mutations cause an enzyme called alpha-galactosidase A (alpha-gal A) to become non-functional or only partially functional.

Decreased activity of alpha-gal A leads to the accumulation inside cells of fatty substances called globotriaosylceramide (Gb3 or GL-3) and lyso-Gb3 that the enzyme would normally break down. This accumulation causes damage in tissues throughout the body. The result is progressive, irreversible organ damage, particularly in the nervous system, heart, and kidneys.

Galafold is what scientists call a chaperone therapy, and is the first of this class of medications. It is designed to restore alpha-gal A activity in patients with what are known as amenable mutations. So far, 269 GLA mutations have been identified as amenable. The medicine does not work in patients with other types of mutations.

Galafold attaches to certain unstable and dysfunctional forms of alpha-gal A, stabilizing the enzyme and partially restoring its activity. This allows the enzyme to go to areas in the cell where it can break down Gb3, reducing the toxic effects caused by Gb3 accumulation.

Galafold in clinical trials

Galafold has been evaluated in two Phase 3 clinical trials covering 127 patients with Fabry disease.

The FACETS study (NCT00925301) assessed its safety and effectiveness in Fabry disease patients with amenable GLA mutations. A key measure of its effectiveness was the number of patients who responded to it. Researchers defined response as a reduction of at least 50 percent in Gb3 deposits in patients’ kidneys. The key finding was that patients with amenable mutations responded to Galafold after six months of treatment.

Amicus’ other Phase 3 study, ATTRACT (NCT01218659), evaluated Galafold’s safety and effectiveness in Fabry disease patients with amenable GLA mutations who had been on enzyme replacement therapy. The main measure of effectiveness was whether patients’ kidney function improved after 18 months of treatment. The key finding was that Galafold was as effective as enzyme replacement therapy in stabilizing kidney function.

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