CHMP recommends Galafold (migalastat ) to treat Fabry disease- Amicus Therapeutics

Amicus Therapeutics announced that the European Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion to approve the oral small molecule pharmacological chaperone migalastat (Galafold) as a first line therapy for Fabry disease in all patients who have an amenable genetic mutation. A final decision from the European Commission (EC) is expected in the second quarter of 2016, after which the Company will begin the country-by-country reimbursement processes. The label approved by the CHMP includes 269 Fabry causing mutations which represent up to half of all patients with Fabry disease.

The benefits of Galafold are its ability to stabilise renal function for up to 18 months in patients with experience of enzyme replacement therapy (ERT) and up to 30 months in ERT-naïve patients. It also improves cardiac function following 18 months of treatment in ERT-experienced patients and 30 months of treatment in ERT-naïve patients. The most common side effect is headache which is seen in more than 10% of patients..

Comment: Galafold was successful against Sanofi's Fabrazyme and Shire's Replagal in two measures of kidney function, a success management saw as evidence that patients can safely switch between the leading enzyme replacement therapies, which require bi-weekly infusions, and migalastat, which is a pill.