Change of tack? FDA backpedals on Amicus NDA, extra safety tests

The company's shares jumped on the news this morning.

After a series of talks with the FDA, Amicus, the biotech at the center of president Donald Trump’s campaign to speed up drug development, can now submit its rare disease drug for review.

This is a reversal from last year when the FDA, which was under different management and a different president, told the biotech that it must do extra safety studies, namely for GI symptoms, for its Fabry disease candidate migalastat that would have pushed back any potential approval by several years.

Now the FDA, under the directorship of Scott Gottlieb (who just this week announced plans to help speed up drug development) said it can submit without the need for costlier and timely tests. The biotech says it is now prepping for an accelerated approval by the fourth quarter.

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“This guidance from the FDA marks a tremendous step forward for thousands of people living with Fabry disease in the United States,” said John Crowley, chairman and CEO of Amicus.

“We are moving ahead expeditiously with our NDA submission and accelerating the U.S. pathway for migalastat. Today is a seminal moment in the development of migalastat and a testament to the dedication and perseverance of the patients, physicians and employees who have worked so hard on the development of this precision medicine.”

Jay Barth, M.D., CMO of Amicus, added: “The FDA’s willingness to review migalastat data reflects what we believe is the gold standard in science-based, data-driven, patient-centric therapeutic development. We believe that we have a robust data package for this NDA submission, and we look forward to advancing toward a planned pathway for U.S. approval for migalastat.”

The spotlight was put onto the company earlier this year when Trump, in his first joint address to Congress in March, made mention of Rare Disease Day and brought into the gallery Megan Crowley, who was diagnosed with the rare condition Pompe disease as a young child.

In a traditional biotech startup story, Crowley’s father John went on to create a new company called Novazyme to help her disease, and is now chairman and CEO of another rare disease biotech, namely Amicus. His startup and its back story also became a 2010 film, Extraordinary Measures, starring Brendan Fraser and Harrison Ford.

Trump used Crowley’s story as an example of the “unbounded power of a father’s love for a daughter,” but then said: “But our slow and burdensome approval process at the [FDA] keeps too many advances, like the one that saved Megan’s life, from reaching those in need.

“If we slash the restraints, not just at the FDA but across our government, then we will be blessed with far more miracles like Megan,” he concluded. It seems as if a new-look FDA, with a focus on speed and approvals, is beginning to take shape.

The biotech has had a tricky path to U.S. approval: It saw a key trial fail back in 2012, which prompted GlaxoSmithKline to later drop out of its partnership with the company. It was then told in 2016 that it couldn’t hope for an approval unless it undertook a new test, but now the path appears much less rocky. Approval is not, of course, guaranteed.

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. Despite being a rare disease, a number of medications are already on the market, including Sanofi Genzyme’s Fabrazyme (agalsidase beta) and Shire’s Replagal (agalsidase alfa).

Migalastat targets a range of mutations in the disease, making it potentially effective in 35% to 50% of the patient population. The biotech told Fiercebiotech in an emailed statement that the FDA "never had concerns over migalastat's safety. There is no known GI side effect of migalastat." The spokesman added: "The additional phase 3 study was initially requested because GI symptoms are one manifestation of the underlying Fabry disease process."

There are around 1,500 Fabry patients in the U.S., of whom around half show GI signs and symptoms, according to recent estimates from analysts at Leerink. 

The company was up as much as 41% premarket this morning on the news. It had a market cap of around $1 billion before Trump’s speech but is now worth around $1.5 billion.