Canadian Agency Urges Reimbursement for Galafold to Treat Fabry Disease

Ana de Barros, PhD avatar

by Ana de Barros, PhD |

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The Canadian Drug Expert Committee (CDEC) has recommended in favor or reimbursement of Galafold (migalastat) for long-term treatment of adults with a confirmed diagnosis of Fabry disease and who have an amenable mutation, Amicus Therapeutics announced.

CDEC will now start conversations with regulatory authorities to make Galafold available to Canadian patients.

“The positive recommendation for Canadian reimbursement reflects an important endorsement of the Galafold value proposition as an oral precision medicine for people living with Fabry disease who have amenable mutations,” John F. Crowley, chairman and CEO of Amicus, said in a press release.

CDEC based its review on Galafold’s clinical development program as well as input from a clinical specialist who treats patients with Fabry disease, and patient group information about outcomes and critical needs of patients and caregivers affected by the disease.

“Following the positive CDEC recommendation, Fabry patients in Canada who have amenable mutations are one step closer to accessing this important oral precision medicine,” said Dr. Daniel Bichet of the University of Montreal, and principal investigator for Canada in the Galafold clinical studies. “Approximately 270 mutations have been identified as amenable to this chaperone therapy in Canada, providing an opportunity for many patients to potentially benefit from this new treatment.”

Galafold works by stabilizing the patient’s dysfunctional alpha-galactosidase A (alpha-Gal A) enzyme, so it can clear the accumulation of disease substrate in people with amenable mutations, or 35 to 50 percent of all Fabry patients. Researchers used a proprietary in vitro assay was used to classify more than 1,000 known GLA mutations as amenable or not amenable to treatment with Galafold. Importantly, Galafold is not recommended for use in patients with a non-amenable mutations.

Amicus, based in Cranbury, New Jersey, is currently recruiting U.S. patients for its expanded access program (NCT01476163), which lets physicians ask Amicus to provide Fabry disease patients access to Galafold. The program allows patients to receive open-label treatment for six months with equal renewable periods.

Migalastat has been approved for use in the European Union under the same brand name, Galafold, for anyone 16 years and older who has Fabry disease with an amenable mutation.