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fter 12 years, millions of dollars, and a major reversal of fortune, Amicus Therapeutics won Food and Drug Administration approval on Friday for its first therapy, a treatment for a rare and sometimes deadly disease.

The drug, called Galafold, targets Fabry disease, an inherited disorder in which the lack of a key enzyme leads to buildups up fats that can cause fatal organ damage. As it stands, Fabry patients rely on costly, bimonthly injections of synthetic enzymes to keep symptoms at bay. Amicus’s drug is an every-other-day pill that works not by replacing the missing enzyme but by boosting the effects of what patients already produce.

The FDA approval clears Galafold for use in patients with certain genetic variants, accounting for up to 50 percent of the roughly 3,000 people with Fabry in the U.S., according to Amicus.

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“It’s a great example of precision medicine, of patient focus, and, frankly, of persistence,” said Amicus CEO John Crowley, who founded the company in 2005.

Just two years ago, Galafold’s potential in the U.S. looked uncertain. Amicus had completed two late-stage trials demonstrating the drug’s positive effects on Fabry. The data were strong enough to convince regulators in Europe, who approved the treatment in 2016. But the FDA wanted more data, demanding Amicus conduct a third study focused on whether Galafold could improve the gastrointestinal symptoms of Fabry. Because the disease is so rare, completing such a study would take a matter of years. Amicus’s stock price dropped by about 30 percent, and Wall Street largely wrote off the drug’s potential in the U.S.

But then things at the FDA began to change. First, in late 2016, President Obama signed into law the 21st Century Cures Act, which, among many provisions, mandated that the FDA step up its interactions with patients and embrace the idea of precision medicine, in which drugs are crafted for specific genetic variants. Then Amicus began a campaign to reach out to FDA leadership about Galafold’s effects on Fabry, Crowley said, presenting new analysis and real-world data gathered from patients in Europe. In early 2017, Crowley even got a private audience with President Trump to discuss the important of focusing on patients with rare diseases.

“It was just night and day,” Crowley said. The FDA “began asking us for case studies, for patient narratives. And at the end of April [of 2017] we provided an enormous briefing book of data.”

Three months later, the FDA had completely reversed course. Rather than requiring a yearslong new study, the agency agreed to consider approving Galafold based on existing data.

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Now, after a saga that saw Amicus gain and lose nearly $2 billion in market value, the company is finally cleared to sell the drug in its home country.

Amicus did not disclose the annual cost of Galafold, but Crowley said it would be less than what patients pay for enzyme replacement, which is the standard of care for Fabry patients. Fabrazyme, the enzyme replacement therapy sold by Sanofi, has a list price of more than $300,000, according to the insurance trade group AHIP. That figure doesn’t include the cost of having the treatment infused, which would not be necessary with the oral Galafold, Crowley said.

And, at a time when the escalating cost of medicines has become a hot-button issue, Amicus is launching its drug with a pledge to never raise the price beyond the rate of inflation, as measured by the Consumer Price Index.

“I think that’s important to show our responsibility as a company to ensure that our medicines are fairly priced and broadly accessible,” Crowley said.

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  • This article fails to point out that the US phase 3 trial failed to hit statistical significance and the EU phase 3 trial had a serious patient imbalance where the Galafold arm had a bunch of patients with a less sick genotype.

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