At Long Last, First Drug Approval in Sight for Amicus

Xconomy New York — 

It’s an understatement to call Amicus Therapeutics’ quest to get its first drug approved a roller coaster ride. But it looks like the twists and turns might soon be over for the Cranbury, NJ-based company—at least in Europe.

The committee of medicinal products for human use (CHMP) on Friday recommended approval of Amicus’s (NASDAQ: FOLD) Fabry disease drug, migalastat (Galafold), meaning there’s a good chance that the drug could soon be approved in Europe. The European Medicines Agency looks to the CHMP for guidance before making a decision on a drug. It’ll now review the CHMP’s opinion, and make a decision on migalastat in the coming months. In a separate statement, Amicus said a ruling is expected during the second quarter.

“This positive CHMP opinion for migalastat is a huge milestone for the Fabry community and a significant step towards our vision to become a leading global biotechnology company focused on rare and devastating diseases,” said Amicus CEO John Crowley (pictured above), in a statement.

Amicus will hold a conference call this afternoon to discuss the news.

Should migalastat win approval, it would be Amicus’s first approved drug, and the first oral treatment for Fabry, a disorder in which cells in the body don’t properly clear out a certain type of fat. The fat builds up in the body and causes potentially serious problems, like progressive kidney damage or heart attacks. There is no cure for Fabry. It’s typically treated with infusions of enzyme replacement therapy, which replaces a missing or faulty enzyme with a genetically engineered, working one. Enzyme replacement therapies like Genzyme’s agalsidase beta (Fabrazyme) and Shire’s agalsidase alfa (Replagal)—the only two Fabry therapies approved in Europe—have to be infused every two weeks.

Migalastat would be geared towards a subset of patients with “amenable [genetic] mutations,” according to the CHMP, representing about 35 to 50 percent of the Fabry population. An estimated 5,000 to 10,000 patients are diagnosed with the disease worldwide.

The CHMP’s ruling is welcome news for Amicus, which has traveled down a long and winding road to get to this point. In 2014 I profiled Amicus’s quest of more than a decade to create “pharmacological chaperones,” or small-molecule drugs designed to grab misfolded enzymes, force them into the correct shape, and shepherd them to the proper cell. Amicus has seen its share of failures trying to prove the worth of this approach, and it looked like the company was finally in the clear in late 2014 after it produced positive results from a Phase 3 study of migalastat. In March of 2015, Amicus announced that it aimed to seek accelerated approval of migalastat in the U.S.—quicker than it had previously expected—following a meeting with the FDA.

That decision backfired. Months later, in October, Amicus said it would no longer file for approval after various “follow-up interactions” with the agency. The FDA wanted more data, and as a result, Amicus faced a delay. Shares plummeted 40 percent, and haven’t recovered since.

In February, Amicus said in a statement that it’s in the process of getting the data the FDA wanted, and expects to meet with the agency in the second quarter, data in hand, to discuss the path forward.

Aside from migalastat, Amicus’s most advanced drug is SD-101 (Zorblisa), a drug in phase 3 testing for a rare, debilitating skin disease called epidermolysis bullosa. Amicus snagged the drug when it bought Durham, NC-based Scioderm in 2015. Data from the study are expected later this year.