Amicus Therapeutics Gets European Commission Approval For Galafold in Patients With Fabry Disease

Amicus Therapeutics FOLD revealed that the European Commission has granted full approval for its oral small molecule pharmacological chaperone Galafold (migalastat). According to the company, it becomes a first line therapy for long-term treatment of adults and adolescents aged 16 years and above with a confirmed diagnosis of Fabry disease (alpha-galactosidase A deficiency) and who have an amenable mutation.

Amicus Therapeutics said that it commenced supplying the Germany market on Monday, May 30 and would start the reimbursement processes with healthcare authorities in each of the big European countries.

The company indicated that Galafold was the first oral treatment and the first precision medicine for Fabry disease. The company added that the broad label included 269 Fabry-causing mutations representing 35 to 50 percent of all patients with Fabry disease. The label also referenced a website www.galafoldamenabilitytable.com where EU healthcare providers could quickly and accurately determine which mutations were amenable to Galafold.

Amicus Chairman and CEO, John Crowley, commented, "This EU approval for Galafold is a significant advancement in the field of precision genetic medicine and a tremendous milestone for the Fabry community. This approval also completes our transformation to a global, fully-integrated, commercial biotechnology company focused on rare and devastating diseases. Our world-class commercial and business leadership team has done an outstanding job preparing for this day. We have begun the launch of Galafold in Germany and will commence the country-by-country reimbursement processes throughout the EU.

He continued, "We are grateful for the ongoing support from our Amicus employees and the Fabry community, in particular those physicians and patients who participated in the clinical studies of Galafold and their families who made this approval possible. As we move forward with the commercial launch of Galafold, we will continue to invest in the innovation of our pipeline. Our vision today is more focused than ever - to build on our strong science and clinical experience to bring forward the highest quality therapies for Fabry, Pompe, EB and other rare and devastating diseases."

The company said the EC approval of Galafold followed the unanimous April positive opinion granted by the Committee for Medicinal Products for Human Use (CHMP) with final authorization pending in Iceland and Norway. The EC approval provided a platform to begin accessing the over 70 percent of the Fabry global market, including the EU member states and several international territories, which accept the EC approval as the basis for marketing submissions.

Amicus said it was also pursuing independent regulatory processes in several international territories outside Europe. This included Japan, Australia and Canada. The company also expects to meet with the FDA in the middle of 2016 to clarify a possible regulatory pathway for Galafold.