Amicus Nabs First Approval as EU Gives Green Light to Fabry Drug

Xconomy New York — 

Amicus Therapeutics is as familiar with the ups and downs of the drugmaking business as anyone. It has dealt with clinical failures, regulatory setbacks, restructurings, and more over a roughly 15-year history. But now the Cranbury, NJ-based company’s big day has finally arrived: Regulators in Europe have approved migalastat (Galafold), the first Amicus drug to make it to market.

Amicus (NASDAQ: FOLD) said migalastat has been approved in Europe as a front-line treatment for certain patients with the rare Fabry disease, a disorder in which cells in the body don’t properly clear out a certain type of fat, which can lead to kidney damage or heart attacks. The drug is specifically approved for Fabry patients with “amenable” genetic mutations, 269 different Fabry-causing mutations that lead to about 35 to 50 percent of cases. An estimated 5,000 to 10,000 patients worldwide are diagnosed with Fabry.

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 is a pill.

Amicus began selling the drug in Germany on Monday and said it will “commence the reimbursement process” with healthcare agencies in each of the major European countries. According to chief operating officer Bradley Campbell, Amicus will price migalastat “roughly at parity with” with ERT, which costs about $300,000 per year. He didn’t provide a specific figure, however.

“We believe this reflects the significant value that [migalastat] brings to patients while potentially giving some savings back to the healthcare systems by avoiding any infusion associated costs,” Campbell said in an e-mail statement to Xconomy.

The decision in Europe was largely a formality ever since the committee of medicinal products for human use (CHMP)—which the European Medicines Agency looks to for guidance before making an approval decision—recommended approval of Amicus’s drug in April. The situation is much more uncertain in the U.S., where the company is currently gathering the data needed to discuss a potential path to approval. Amicus had said in March 2015 that it aimed to seek accelerated approval of migalastat in the U.S., but abruptly changed course months later after various “follow-up interactions” with the agency. Today, Amicus said that it aims to meet with the FDA in the middle of 2016 to clarify migalastat’s future in the U.S. Amicus will also seek approval of migalastat in Japan, Australia, and Canada.

Amicus was formed in 2001. Here’s more on its long and winding road to create “pharmaceutical chaperones,” small-molecule drugs designed to grab misfolded enzymes, force them into the correct shape, and shepherd them to the proper cell.