Amicus Therapeutics, Inc. (NASDAQ:FOLD) witnessed a sharp increase in its volume after the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) backed the company’s migalastat for Fabry disease.
The Committee found the drug appropriate as a first-line therapy for patients with the amenable genetic mutation. However, the European Commission (EC) will take the final call only during the second quarter of 2016. Approval from the EC will take forward Amicus to start reimbursement processes country-by-country.
The CHMP has approved the label for the drug for nearly 269 mutations stemmed from Fabry. Such approval represents nearly 50% of the patients impacted by Fabry disease. On receiving the positive opinion from the CHMP, Amicus’ Chairman and CEO, John F. Crowley, said that the Committee’s opinion serves as a considerable milestone for both the Fabry disease-impacted patients and the company.
269 mutations referred
He went on to state that the label approved by the CHMP is broad and reflects nearly 35% to 50% of the Fabry community. Thus, it represents a significant opportunity for the company to address the treatment needs of a wide Fabry cases. The company expressed its gratitude towards Fabry patients, who chose to participate in the clinical studies of migalastat.
Following the receipt of the positive opinion from the CHMP, the company is set to spearhead its launch related activities across countries in Europe. Meanwhile, Prof. Dominique P. Germain, MD, Ph.D., Division of Medical Genetics at the University of Versailles and Assistance Publique, regards the positive opinion as a successful event for the company and places it on the track to gain EC’s nod.
The Committee has recommended the use of the drug in adults and adolescents aged 16 years and more. The stock of Amicus gained 2.84% to $8.69 during the previous day’s trading session.