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The Food and Drug Administration has changed its tune on an experimental drug for a deadly rare disease, withdrawing a request that the company developing it run another clinical trial. The unusual move comes after President Trump met with the company’s CEO — and promised to speed up what he called a “slow and burdensome” process for drug approvals.

Amicus Therapeutics, a New Jersey biotech company, announced on Tuesday morning that the FDA had given it the all-clear to submit for review its treatment for Fabry disease, an inherited disorder that often leads to fatal organ damage. The FDA is not guaranteeing that the drug will be approved, but is signaling that regulators believe the data are strong enough to merit consideration. That’s a reversal: Just last year, the FDA asked Amicus to do another study on side effects, which would have delayed approval by two years.

Now, Amicus says it’s on track to apply for approval by the end of the year. Amicus CEO John Crowley told STAT that if all goes well, the drug, Galafold, could reach the U.S. market in the second half of 2018. It’s already on the market in Europe.


Crowley has been actively and very publicly pressing the FDA to move faster to get rare disease treatments to market.

In February, he met with the president to discuss the issue. Later that night, Crowley’s daughter, Megan, who has a rare disorder called Pompe disease, sat in the first lady’s box during Trump’s first address to a joint session of Congress. Trump hailed Megan by name and promised to speed treatments for other rare conditions.


“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,” Trump said.

Crowley, who has been repeatedly mentioned as a possible Republican candidate for Senate, dismissed the idea that Trump’s rhetoric had any bearing on the FDA’s Amicus decision.

In his mind, the FDA is in the midst of “an evolution” rather than a “seismic shift,” one driven as much by the bipartisan 21st Century Cures Act signed by President Obama as by Trump’s prodding.

“I don’t believe at all politics played a role in this,” Crowley said. “I wish it were so easy.”

Amicus has been developing Galafold for more than a decade and, as recently as 2015, looked to be on the path to FDA approval for the drug. Then regulators took issue with Amicus’s supporting data, requesting an entirely new trial to flesh out Galafold’s effects on the gastrointestinal symptoms of Fabry.

The U.S. delay scrambled Amicus’s timeline, tanked its stock price, and left Crowley frustrated.

European regulators apparently saw no problem with Galafold’s safety, approving the drug in 2016. But “the FDA has declared that the only pathway to availability for Fabry patients in the United States is predicated on yet another study,” Crowley wrote in a March essay published by the Observer, which is owned by Trump’s son-in-law, Jared Kushner. “And while we do that, patients wait. All get sicker. And some will die.”

Fabry affects about 3,000 people in the U.S., according to Amicus. Galafold is intended treat the roughly 50 percent of those patients who have certain genetic mutations. The list price in Europe is about $260,000 a year, according to Biocentury.

The FDA’s decision on Amicus comes amid a whirlwind few months for the new commissioner, Scott Gottlieb. Since taking office in May, Gottlieb has repeatedly pledged to speed up the path of new treatments for serious diseases, announcing a flurry of initiatives aimed to make the agency more flexible.

  • I am so impressed with on going progress to approval of the drug treatment for Megan and 3K possible subscribers to it.
    Congratulations, John & Megan Crowley for guiding the Political system in the right direction.
    It is better to monitor gastric impact to each patient during rare disease treatment than to wait while affecting organs and ultimately inducing death.
    If logic and monitoring could be applied to all potential drugs and side effects it would be a less expensive win/win situation.

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