Amicus Files NDA for Fabry Disease Drug

Published on: 

The company is seeking approval from FDA for use of the drug, migalastat, to treat Fabry disease in patients who have amenable mutations.

On Dec. 14, 2017, Amicus Therapeutics, a biotechnology company, announced that it has submitted a new drug application to FDA requesting approval for migalastat HCl, an oral precision medicine for treating patients 16 years and older with Fabry disease who have amenable mutations.

Migalastat previously received both orphan drug designation and fast track designation from FDA. The drug works by stabilizing the body's own dysfunctional alpha-Gal A enzyme, so that the accumulated disease substrate in patients who have amenable mutations can be cleared, according to the company.

The drug, under the trade name Galafold, was granted full approval in the European Union (EU) by the European Commission as a first line therapy for long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease (alpha-galactosidase A deficiency) and who have an amenable mutation. Outside the EU, migalastat is approved in Switzerland, Israel, Australia, and Canada, with regulatory submissions under review in Japan and additional geographies.


John F. Crowley, chairman and CEO of Amicus Therapeutics, stated in a company press release, "Today marks the very first Amicus submission of a new drug application to [FDA]. This important milestone is the culmination of a strong collaboration and commitment among the patients, physicians, and Amicus employees who have spent more than a decade to advance migalastat. On the heels of our initial launch success for migalastat in Europe, our goal is to further expand access for more Fabry patients with amenable mutations in the US, Japan, and other global geographies.  We look forward to working with [FDA] during the review process and to a potential US approval of migalastat in 2018."

Source: Amicus Therapeutics