FDA permits Amicus to file for Fabry disease drug migalastat, reversing previous stance

Shares in Amicus Therapeutics jumped as much as 40 percent Tuesday after the company said the FDA has agreed to review a new drug application for the Fabry disease drug migalastat. According to Amicus, the decision reverses the FDA's stance from last November, when the agency denied the company's request to seek accelerated approval of the oral chaperone therapy based on data it had accumulated on reductions in the globotriaosylceramide (GL-3) biomarker.

Amicus, which now plans to file the application for accelerated approval in the fourth quarter, said it will base its submission "on existing data, including reduction in disease-causing substrate GL-3, as well as the totality of data from completed clinical studies." The FDA had declined to consider Amicus' application last year in part because one of two clinical migalastat trials had failed to meet its main goal. According to CEO John Crowley, the company convinced the FDA to examine its data using new analyses, as well as patient testimonies, saying "we didn't ask them to lower the bar, we just asked them to review the existing data."

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The FDA had also requested that Amicus conduct an additional Phase III trial of migalastat to assess gastrointestinal (GI) symptoms in Fabry disease patients who have an amenable mutation, with those results expected to have been available in 2019. However, now the company says the GI study is "no longer required" prior to its submission.

Migalastat was approved last year by European regulators under the brand name Galafold as monotherapy for the long-term treatment of Fabry disease in adults and adolescents aged 16 years and older with amenable mutations. Jay Barth, chief medical officer at Amicus, noted that "the data from our clinical trials, including the two largest pivotal studies ever completed in Fabry disease, have already supported approvals for migalastat in the EU, Israel and Switzerland, as well as our pending regulatory submissions in Japan, Canada and Australia." He added "we believe that we have a robust data package for [the US] submission."

For related analysis, read ViewPoints: Amicus benefits from high-quality data, or falling FDA standards?

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