Amicus Therapeutics Receives Positive CHMP Opinion for Approval of Migalastat in Patients with Fabry Disease in European Union

Amicus Therapeutics (NASDAQ:FOLD), a biotechnology company at the forefront of rare and orphan diseases, today 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 as a first line therapy for Fabry disease in all patients who have an amenable genetic mutation.
According to the news:

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.

John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. commented:

This positive CHMP opinion for migalastat is a huge milestone for the Fabry community and a significant step towards our vision to become a leading global biotechnology company focused on rare and devastating diseases. Oral migalastat represents a groundbreaking approach to personalized medicine. We are very pleased that the CHMP has adopted a positive opinion to approve migalastat with a broad label that includes all 269 amenable mutations screened through our Amenability Assay, as well as a fully searchable Amenability website for physicians. These amenable genetic mutations are represented in an estimated 35% to 50% of the diagnosed Fabry population today, which is a significant opportunity for Amicus to deliver a therapy option for many patients in need. We are grateful for the ongoing support from the Fabry community to advance migalastat, in particular those patients who participated in the clinical studies of migalastat and their families. Our world-class commercial and business leadership team is already in place in key launch countries in Europe, and we look forward to a final EC decision in the coming months. We are fully prepared and sharply focused on the launch of migalastat to complete our transformation into a global commercial organization.

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