FDA grants accelerated approval for new oral treatment for Fabry disease

The US Food and Drug Administration (FDA) has granted accelerated approval for a new treatment for adults with Fabry disease — a rare genetic disorder, resulting from the build-up of globotriaoscylceramide (GL-3) in blood vessels and organs.

Galafold (migalastat) is the first oral medication approved for the treatment of this disease and is indicated for adults with Fabry disease who have a genetic mutation determined to be responsive to treatment with Galafold based on laboratory data.

It was approved under the Subpart H Accelerated Approval pathway based in reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate. As a condition of its approval, the manufacturer (Amicus Therapeutics) will continue to study the drug in a confirmatory Phase IV programme.

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

“This FDA approval of Galafold is a transformative moment for people in the US living with Fabry disease, as it gives adult patients with amenable GLA variants a new treatment option for the first time in more than 15 years,” stated John F. Crowley, chairman and chief executive officer of Amicus Therapeutics. “The Fabry disease community has had an active voice in every stage of development of this medicine. We are grateful to this wonderful and passionate community, particularly the patients and physicians who have made this research possible through their participation in the clinical trials, as well as to the US regulators and our ever-persistent and dedicated Amicus team.”

“[It has been] a long-awaited day of celebration for all of us living with and advocating for people with Fabry disease, especially those who have participated in the development of Galafold in the US,” added Jack Johnson, founder and executive director, Fabry Support & Information Group. “With the FDA approval of Galafold, certain members of the US Fabry disease patient community finally have a second treatment option.”

This approval was based on data from a Phase III study in treatment-naïve patients — Study 011 or FACETS. In this study the efficacy of Galafold was demonstrated with patients experiencing a reduction in the amount of damaging disease substrate accumulated in the kidney capillaries.