With Data in Hand, Amicus Preps For Drug Filings in U.S., Europe

Xconomy New York — 

Back in 2013, Amicus Therapeutics was down in the dumps. It shelved an FDA application for its lead drug, a pill for a rare condition called Fabry Disease, and faced a long road—two trials to finish, and a lot of data to accrue.

Two years later, Amicus has made it just about all the way back. The Cranbury, NJ-based company plugged away, gathered data, and now it’s about to ask regulators in the U.S. and Europe to at long last approve its drug.

Amicus (NASDAQ: FOLD) provided two updates on the regulatory front this morning. First, it’ll file papers for approval of its Fabry pill, migalastat, in Europe, in the second quarter. Second, based on feedback from the FDA at a meeting earlier this week, Amicus intends to file an application for U.S. approval in the second half of the year.

Amicus said the FDA application would come under the FDA’s “accelerated approval” pathway, which is meant for drugs for serious conditions with few therapeutic options available. The FDA, Amicus said, is open to considering various “surrogate” measures for approval—like improvements in certain statistical measures of kidney function. These “surrogate” measures might suggest earlier on if the drug is helping patients, rather than if Amicus were to wait for clear clinical benefits.

Amicus and the FDA have also discussed the parameters of a confirmatory study that would be run post-approval; such trials are required under the accelerated approval pathway.

The company will hold a conference call this morning to discuss the news. Amicus shares surged more than 20 percent in pre-market trading.

The regulatory updates are the latest step on a long, winding road for Amicus, a journey I profiled in Xconomy last year. Launched in 2002, the company has spent more than a decade working on “pharmacological chaperones,” or small-molecule drugs designed to grab misfolded enzymes, force them into the correct shape, and shepherd them to the proper location in a cell. These drugs are meant for lysosomal storage disorders, which are caused by mutations in the genetic instructions for important enzymes that normally help our cells clear out waste like extra fats and sugar.

Amicus has seen its share of failures trying to prove the worth of this approach. A drug for Gaucher’s failed in 2012. Then, migalastat missed its goal in the first stage of a Phase 3 study in 2012, and Amicus pulled an FDA application for the drug several months later and lost GlaxoSmithKline as a partner (the British firm stayed on as an Amicus investor).

But Amicus dug through the data, found that patients with certain mutations (estimated to be about 30 to 50 percent of Fabry patients) were responding better, and convinced the FDA to change the clinical goals for its trial. It had to restructure and gamble on migalastat to succeedas CEO John Crowley told me last year, he had to convince Amicus’s board to “double down” on the drug—but the company has since accrued data supporting its thesis. Soon it’ll find out if those data are good enough for regulators.

“These regulatory discussions demonstrate that health officials in both the U.S. and [Europe] recognize the continued life threatening medical challenges that people living with Fabry face each day,” Crowley said in a statement. “It has been a stellar example of industry and regulators working together to evaluate data and to determine the best and fastest ways in which to bring novel therapies to people in need, especially in such a rare and devastating disease like Fabry.”