Amicus found the drug effective in patients after 12 months of treatment in the trial, which tested migalastat as a form of monotherapy in patients with Fabry disease, a disorder in which a fat called globotriaosylceramide builds up in the body's cells. The company also released positive 24-month data.
"Today is a great day for Amicus and the Fabry community," said Chairman and CEO John F. Crowley in a statement. "Pending positive data from our second Phase 3 study we expect to meet with regulatory authorities to discuss these data and determine the fastest registration pathway for migalastat."
The stock was up 21.74% to $2.24 at 2:04 p.m.