Amicus Therapeutics, Inc. Launches Galafold for Treatment of Fabry Disease in Spain
January 17, 2018 at 09:01 pm
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Amicus Therapeutics has commenced the commercial launch of the precision medicine Galafold in Spain following final pricing and reimbursement decisions. Galafold is now reimbursed in Spain 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. Fabry disease is a genetic disease which causes deficiency of an enzyme called alpha-galactosidase A (alpha-Gal A), which is the result of mutations in the GLA gene. As a precision medicine, Galafold is designed to restore alpha-Gal A activity in patients who have amenable mutations (an estimated 35% to 50% of the Fabry population). The European Commission granted the first approval for Galafold for 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. Outside the EU, migalastat is approved in Switzerland, Israel, Australia, Canada and South Korea, with regulatory submissions under review in additional geographies.
Amicus Therapeutics, Inc. is a biotechnology company, which is focused on discovering, developing, and delivering novel medicines for rare diseases. Its two marketed therapies are Galafold, the first oral monotherapy for people living with Fabry disease who have amenable genetic variants, and Pombiliti + Opfolda, a novel treatment designed to improve uptake of active enzyme into key disease relevant tissues for adults living with late-onset Pompe disease. As an orally administered monotherapy, Galafold is designed to bind to and stabilize an endogenous alpha-galactosidase A (alpha-Gal A) enzyme in those patients with genetic variants identified as amenable in a Good Laboratory Practice (GLP) cell-based amenability assay. Pombiliti + Opfolda consists of a uniquely engineered rhGAA enzyme, cipaglucosidase alfa-atga, with an optimized carbohydrate structure to enhance lysosomal uptake, administered in combination with miglustat that functions as an enzyme stabilizer.