US biotech Amicus Therapeutics has launched its new Fabry disease treatment Galafold (migalastat) in Italy after winning reimbursement coverage.
Final reimbursement guidelines from the Ministry of Health back Galafold for long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease (alpha-galactosidase A deficiency) and who have an amenable mutation.
John Crowley, Amicus chairman and CEO, said: “The commercial launch of Galafold in Italy is a significant milestone for the Fabry community.
“We believe that our successful pricing and reimbursement in Italy is a testament to the significant value of our oral precision medicine, and reflects our growing momentum with the country-by-country processes to launch Galafold throughout the EU.”
Galafold is the first orally administered alternative to the current crop of injectable enzyme replacement therapies (ERT) for Fabry disease, such as Sanofi’s Fabrazyme (agalsidase beta).
Amicus’ therapy is a first-in-class chaperone therapy and works by stabilising the body’s own dysfunctional enzyme.
Galafold received NICE backing for England and Wales last month and has been recommended as a treatment option in the US, on condition that Amicus provide it at a discounted rate.