FDA Gives Galafold Accelerated Approval for Treatment of Fabry Patients

The U.S. Food and Drug Administration has granted accelerated approval to Galafold (migalastat) 123 mg capsules to treat adults with a confirmed diagnosis of Fabry disease who have an amenable galactosidase alpha gene (GLA) mutation.

“This FDA approval of Galafold is a transformative moment for people in the U.S. 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,” said John F. Crowley, chairman and CEO of Amicus Therapeutics, which developed Galafold.

Fabry disease is caused by mutations in the GLA gene, which lead to a defective alpha-galactosidase A enzyme. As a result, the enzyme cannot break down globotriaosylceramide (GL-3), a type of fat, that then builds up in the lysosomes of patients’ cells.

Galafold restores enzymatic activity and clears GL-3 buildup, and is suitable for patients who have a confirmed diagnosis of Fabry disease and a GLA mutation amenable to the therapy. Currently, amenable mutations account for 35 to 50 percent of all Fabry patients worldwide. The FDA has approved Galafold for 348 amenable GLA variants.

“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 U.S. regulators and our ever-persistent and dedicated Amicus team. With our new and highly motivated U.S. leadership team, we are poised to make Galafold available to as many appropriate patients as possible,” Crowley said in a statement.

Galafold was approved under the subpart H accelerated approval pathway, which applies to new medicines that have shown safety and effectiveness in treating serious or life-threatening diseases and provide meaningful therapeutic benefit over available therapies.

The approval was based on clinical data from the Phase 3 FACETS clinical trial (NCT01218659) that compared the drug’s safety and effectiveness to  enzyme replacement therapy (Fabrazyme, Replagal) in adults with Fabry disease who had GLA mutations.

The results demonstrated Galafold’s effectiveness, with a reduction in the amount of GL-3 in a specific type of kidney cells called kidney interstitial capillary cells.

The most frequent adverse events included headache, urinary tract infection, nasopharyngitis (a common cold), nausea and fever.

Amicus will continue to study this treatment in a confirmatory Phase 4 program.

“Today is 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 U.S.,” said Jack Johnson, founder and executive director of Fabry Support & Information Group.

“With the FDA approval of Galafold, certain members of the U.S. Fabry disease patient community finally have a second treatment option,” Johnson said. “Through their unwavering commitment and scientific innovation, Amicus has provided a much-needed new treatment option for many Fabry patients.”

In February 2018, the FDA granted Galafold priority review, accelerating its clinical development program.