Yes, Call It a Comeback: Amicus Fabry Drug Posts Big Study Win

CRANBURY, NJ ( TheStreet) -- Amicus Therapeutics ( FOLD) CEO John Crowley came close to permanently shelving his company's experimental Fabry disease therapy following disappointing results from a late-stage study in December 2012 and the departure of partner GlaxoSmithKline ( GSK) about one year later. But Crowley -- famous for his perseverance -- decided to stick with the drug, migalastat, in the hope that a slower-to-enroll, second phase III study would resurrect chances for eventual approval.

Crowley made the right call. On Wednesday, Amicus announced results from the study demonstrating that Fabry patients taking currently available injectable drugs could switch successfully to migalastat, a pill taken every other day, and maintain kidney function required to keep the rare, inherited disease under control. 

Based on the positive study results, Amicus intends to seek European approval for migalastat early next year. The company's U.S. filing plans are less clear. Amicus will schedule a meeting with FDA officials as soon as possible to determine if Wednesday's study results, plus data from the previous phase III study, are sufficient to seek migalastat approval here.

"We came very close to stopping development of migalastat, but we always believed patients were benefitting from the drug, so we tripled down on the program," said Crowley in a phone interview Tuesday night. 

If approved, migalastat would be the first oral therapy for Fabry, giving it a significant convenience factor over Sanofi's ( SNY) Fabrazyme and Shire's ( SHPG) Replagal, both of which require bi-weekly intravenous infusions. Sales of Fabrazyme and Replagal, combined, total about $1 billion per year. Unlike its competition, Migalastat only works in Fabry patients with a specific genetic mutation, estimated to encompass between 30% and 50% of Fabry patients worldwide. 

Amicus shares closed Tuesday night at $4.57.

Fabry is an inherited disease affecting about 10,000 people worldwide in which a genetic mutation stops an enzyme from breaking down a fatty substance known as globotriaosylceramide, or GL3. The buildup of GL3 in blood vessels throughout the body causes severe damage to kidneys, heart, brain, and other organ systems. Current Fabry treatments Fabrazyme and Replagal replace the patient's misshapen or missing native enzyme and clears GL3 from the body. 

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