Following final regulatory adjustments, Galafold is now reimbursed in Spain for age-eligible patients with a confirmed diagnosis of Fabry disease and who have an amenable mutation, which is 35 to 50 percent of the Fabry population.
In Fabry disease, the defective alpha-galactosidase A enzyme leads to buildup of globotriaosylceramide (GL3) lipids throughout the body, including kidney cells.
Galafold is a precision medicine that works by restoring the alpha-galactosidase enzyme’s activity in patients carrying amenable mutations in the GLA gene.
“The commercial launch of Galafold in Spain adds to the tremendous momentum in expanding our launch throughout the EU, where we have now secured reimbursement in all five countries that have the largest Fabry populations within the EU,” John F. Crowley, chairman and CEO of Amicus, said in a press release. “We believe that our pricing and reimbursement success throughout the EU, in addition to our global progress in securing additional approvals and completing new regulatory submissions, are a testament to the significant value of migalastat as the first oral precision medicine for people living with Fabry disease who have an amenable mutation.”
The precision medicine was approved in 2016 by the European Medicines Agency (EMA) for anyone 16 years and older who has Fabry disease with an amenable mutation. Migalastat has additional approvals outside the EU, including Switzerland, Israel, Australia, Canada and South Korea, and has approvals pending in several other geographies.
The U.S. Food and Drug Administration (FDA) received Amicus’ new drug application (NDA) for migalastat (the active ingredient in Galafold) in January 2018. The NDA seeks migalastat’s approval in the U.S. as an alternative to intravenous enzyme replacement therapy. The FDA already has granted migalastat orphan drug and fast track designations.
Amicus is recruiting 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.
Up to 20 patients worldwide may be treated. However patients must meet specific criteria, including age (16-74 years old), confirmed GLA gene mutation shown to be responsive to migalastat, and have no treatment option because either unsuitable for enzyme replacement therapy (ERT) or unable to access ERT. Also, participants must meet requirements for sufficient kidney function.
In Canada, the country’s Drug Expert Committee (CDEC) recently recommended in favor of reimbursement of Galafold for long-term treatment of adults with a confirmed diagnosis of Fabry disease and who have an amenable mutation. The CDEC will start conversations with regulatory authorities to make Galafold available to Canadian patients.
Healthcare providers in the European Union may access this website to identify which mutations are categorized as “amenable” to Galafold. Amicus anticipates submitting additional updates to the label as more GLA mutations are identified and tested.