Amicus Therapeutics received European Commission approval for Galafold (migalastat) as a first-line therapy for the long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease and who have an amenable mutation, the company reported Tuesday. According to Amicus, Galafold "is the first oral treatment as well as the first precision medicine for Fabry disease."
Amicus noted that it launched Galafold in Germany on May 30, and will start the reimbursement processes with healthcare authorities in each of the major European countries. Chief operating officer Bradley Campbell explained that the company will likely price the product "roughly at parity with" enzyme replacement therapy without providing a specific figure. "We believe this reflects the significant value that [Galafold] brings to patients while potentially giving some savings back to the healthcare systems by avoiding any infusion associated costs," Campbell added.
The approval of Galafold, which follows a positive recommendation  by the European Medicines Agency's Committee for Medicinal Products for Human Use last month, was supported by data from two Phase III studies. Amicus indicated that in Study 011 , the therapy was shown to substantially reduce kidney interstitial capillary globotriaosylceramide levels in treatment-naïve patients, while in Study 012 , the drug demonstrated similar efficacy as Sanofi's Fabrazyme (agalsidase beta) and Shire's Replagal (agalsidase alfa) in patients who were previously treated with the enzyme replacement therapies.
Amicus said that regulatory submissions are anticipated for several markets outside Europe, including Japan, Australia and Canada, while the drugmaker plans to meet with the FDA later this year to discuss a regulatory path forward for Galafold in the US. The company disclosed  last year that a US filing for the treatment would likely be delayed after the FDA requested additional information from clinical studies.