After FDA about-face, Amicus' Galafold nabs Fabry disease nod to challenge Sanofi

Once thought unlikely to reach the U.S. until 2020 because of an FDA roadblock, Amicus Therapeutics’ Fabry disease drug Galafold won a green light Friday. Now, to duel with Sanofi's standard therapy Fabrazyme, it's arming itself with a lower price.

The approval, for adults with certain variants of a rogue gene, comes less than a year after the FDA backtracked on previous demands for a new safety trial and put Galafold on an accelerated approval track. 

As the first oral Fabry drug—and the first to treat the disease by boosting the body's own missing enzyme rather than replacing it—Galafold will aim to grab market share from Fabrazyme, which brought in €722 million ($824 million) for Sanofi last year.

And it will do so with a price of $315,000 a year, a small discount to standard enzyme replacement therapies like Fabrazyme, Amicus CEO John Crowley said on a conference call Monday morning. According to Crowley, based on the average weight of adult patients in its U.S. clinical studies of Galafold, the price of Fabrazyme would be $344,000.

Crowley promised the company would never raise Galafold's price tag more than the consumer price index and pledged "a meaningful portion" of Galafold sales to fund R&D specifically for Fabry until there’s a cure.

Galafold's path to U.S. approval hit a snag two years ago, when the FDA flagged concerns about its potential gastrointestinal side effects and asked Amicus to run a new GI safety trial. At the time, Amicus figured it wouldn't have the data ready until 2019.

But under the newly minted commissioner Scott Gottlieb, the agency reversed its decision on Galafold and agreed to consider the drug without the additional data. That was a few months after President Donald Trump, in his first joint address to Congress, brought in Crowley’s daughter Megan, who suffers from the rare condition Pompe disease. During his speech, Trump blasted the FDA’s previous "slow and burdensome approval process" for delaying many medical advances.

The FDA approved Galafold under its accelerated approval pathway based on a phase 3 trial that showed the drug reduced fat in blood vessels in the kidneys. Amicus is required to conduct a phase 4 trial to verify its clinical benefits.

The company said it will launch Galafold immediately, and the company’s Chief Operating Officer Bradley Campbell said on Monday’s call that it already has a full sales and marketing team ready. For 2017, Amicus expects around $90 million sales from the drug, with the addition of Japan and the U.S.

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