Amicus' rare disease drug lines up for a quick EU approval

A depiction of Amicus' chaperone technology for enzyme replacement therapy--Courtesy of Amicus

Amicus Therapeutics ($FOLD) has landed on the European regulatory fast track, potentially shortening the review time for its orphan drug as it prepares filings at home and abroad.

European regulators have granted an accelerated assessment to migalastat, Amicus' treatment for the rare genetic disorder Fabry disease. The company is on track to submit the drug this quarter, management says, and the European Medicines Agency's promise could slash migalastat's review time from the standard 210 days to just 150 days.

Meanwhile, after a March meeting with the FDA, Amicus expects to file a U.S. application for its lead drug in the second half of this year, angling for an accelerated approval through the agency's pathway for treatments that address serious unmet needs.

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

Migalastat's current clear to path to regulators represents a major change of fortune for Amicus, which watched its market value plummet in 2012 when the drug failed to beat placebo in a pivotal trial. That misstep sent partner GlaxoSmithKline ($GSK) out the door and appeared to signal the end for the program, but Amicus persevered, last year 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. Since then, the drug has been a clinical success.

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