European Commission approves Galafold (migalastat) as a treatment for Fabry disease.- Amicus Therapeutics

Amicus Therapeutics has announced that the European Commission has granted full approval for the oral small molecule pharmacological chaperone Galafold (migalastat) as a first line therapy 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. Amicus began supplying the market in Germany on Monday, May 30, 2016 and will commence the reimbursement processes with healthcare authorities in each of the major European countries.

Galafold is the first oral treatment as well as the first precision medicine for Fabry disease. The broad label includes 269 Fabry-causing mutations which represent 35 percent to 50 percent of all patients with Fabry disease.

Comment: In its Phase III program, Galafold measured up to Sanofi's Fabrazyme and Shire's Replagal in two measures of kidney function, which management saw as evidence that patients can safely switch between the leading enzyme replacement therapies, which require biweekly infusions, and migalastat, which is a pill.