Amicus Therapeutics announced Friday that the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recommended its oral small molecule pharmacological chaperone Galafold (migalastat) as a first-line therapy for Fabry disease in all patients with an amenable genetic mutation. The company noted that the label approved by the CHMP includes 269 Fabry-causing mutations, which represent up to half of all patients with the disease. Amicus' shares jumped as much as 10 percent on the news.
Specifically, Amicus noted that the proposed indication is for long-term treatment of patients aged 16 years and older with a confirmed diagnosis of Fabry disease and an amenable mutation. CEO John F. Crowley remarked "oral migalastat represents a groundbreaking approach to personalised medicine."
The CHMP opinion was based on Phase III study data in treatment naïve patents in Study 011 and enzyme replacement therapy switch patients in Study 012, as well as ongoing long-term extension studies, Amicus said, adding that a final decision from the European Commission is expected in the second quarter.
Last October, Amicus announced that it was unlikely to apply for US approval of migalastat by the end of 2015, as it had previously suggested it would do, as the FDA asked the company for more information from clinical trials.
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