Amicus eyes a 2015 EU filing for its rare disease drug with FDA in sight

Amicus Therapeutics CEO John Crowley

Amicus Therapeutics ($FOLD) says it's on track to file its Fabry disease treatment for European approval next year, the next major step for a biotech in the midst of comeback.

Following a positive presubmission meeting with the European Medicines Agency, Amicus is now preparing its application and says it's on track to submit its drug, migalastat, in the middle of 2015. If the EMA accepts the filing, it'll kick off a review period that generally takes around 210 days, according to regulators, giving Amicus the chance to hit the European market as early as 2016. In the U.S., the company is slated to meet with the FDA in the first quarter of next year to hammer out a regulatory path in its home country.

Migalastat is designed to unseat the standard-of-care enzyme-replacement therapies (ERTs) currently used to treat Fabry, which results from a deficiency of alpha-galactosidase A and often leads to renal failure and death. In pivotal trials, the drug measured up to Sanofi's ($SNY) Fabrazyme and Shire's ($SHPG) Replagal in two measures of kidney function, meaning patients can safely switch between the leading ERTs, which require bi-weekly infusions, and migalastat, which is a pill.

The drug's latest Phase III success was a welcome reversal of fortune for Amicus, which lost deep-pocketed partner GlaxoSmithKline ($GSK) and a major chunk of its market cap after migalastat failed to beat placebo in a 2012 pivotal trial. But CEO John Crowley and his team never gave up on the treatment, convincing the FDA to consider a different biomarker for improving Fabry symptoms and to evaluate migalastat solely in the roughly 30% to 50% of patients who carry a specific cellular mutation. Through that lens, the drug has been a clinical success, and Amicus believes it's on the path to global commercialization.

"The submission of our marketing application in Europe will be an important milestone in our global strategy to get migalastat approved for Fabry patients with amenable mutations as quickly as possible," Crowley said in a statement. "We believe significant unmet need still exists in the treatment of Fabry disease, and that migalastat could become a very important, differentiated oral therapy."

Beyond its lead candidate, Amicus is at work on a next-generation treatment that combines enzyme replacement with the chaperone technology at play in migalastat, nearing the clinic with a therapy that could make a difference for the patients not eligible for its other Fabry drug.

- read the statement

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