Blocked in U.S., Amicus gets a green light for Fabry drug in Europe

EU symbol

Amicus Therapeutics has followed through on its promise of a European approval for its Fabry drug migalastat (Galafold), clawing back some of the market respect lost in last fall’s surprise retreat on the FDA front.

The Cranbury, NJ-based biotech says it gained full approval from the European Commission and started supplying Germany yesterday. The rest of the European markets are now being targeted in the first commercial rollout for the biotech.

Shares of Amicus ($FOLD) jumped 7% on the news, which was expected by most analysts covering the company.

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Back in early October of 2015, the company did a surprising about-face maneuver at the FDA. After signaling to investors that its pre-NDA meeting with the agency had gone well, the biotech’s executive team did a careful read of the meeting notes, reviewed some follow-up communications and concluded that they had been seriously overconfident. Not only did they need to do more work on the existing data, the company raised the prospect of tacking on a new trial, which would seriously delay any marketing approval in the U.S.

The 15-year-old Amicus has been on a roller-coaster ride for years now, watching a key trial fail in 2012, seeing GlaxoSmithKline ($GSK) drop out of its partnership and then picking up the pieces with a new surrogate endpoint to consider. Amicus will now target more ex-U.S. approvals as it tries to hammer out a pathway forward at the FDA over the course of this year.

Fabry disease is a genetic lysosomal storage disease caused by an enzyme malfunction that allows a type of fat called globotriaosylceramide, GL-3, to accumulate in cells. Migalastat targets a range of mutations in the disease, making it potentially effective in 35% to 50% of the patient population.  

"This approval also completes our transformation to a global, fully-integrated, commercial biotechnology company focused on rare and devastating diseases,” noted Amicus CEO John Crowley in a statement.

- here's the release

Related Articles:
On second thought, Amicus won't be filing its lead drug this year
Amicus plots an end-of-year FDA filing for its resuscitated lead drug
Amicus' rare disease drug lines up for a quick EU approval

Image: Lars Aronsson

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