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Alexion's Ultomiris gets US FDA approval for ultra-rare kidney disease

Alexion Pharmaceuticals Inc. said the U.S. Food and Drug Administration approved its drug Ultomiris for treating atypical hemolytic uremic syndrome, an ultra-rare kidney disease.

The U.S. regulator previously granted priority review to Ultomiris, or ravulizumab-cwvz, for the treatment of atypical hemolytic uremic syndrome, or aHUS, in order to inhibit complement-mediated thrombotic microangiopathy in adult patients and pediatric patients one month of age and older. The condition causes abnormal blood clots to form in small blood vessels, commonly in the kidneys, which restrict or block blood flow.

AHUS is caused by chronic, uncontrolled activation of the complement system, a part of the body's immune system that destroys and removes foreign particles. The disease can lead to progressive damage to other vital organs in the body as well.

This is the first pediatric approval for the Alexion therapy. The company has filed applications for Ultomiris for the treatment of aHUS in the EU and Japan as well.

The FDA approval was supported by data from two trials that are part of a broader phase 3 study, which showed the drug improved kidney function and platelet count in patients with the disease.

The regulatory approval came days after Alexion acquired Achillion Pharmaceuticals Inc. for an initial payment of $930 million — a deal described as "high risk, high reward" by analysts.

Boston-based Alexion is touting Ultomiris as an improved version of its older drug Soliris. The drugmaker has thrown its weight behind promoting ravulizumab as the patent for Soliris in Europe is set to expire in 2023.

Ultomiris is approved in the U.S., EU and Japan to treat a rare blood disease called paroxysmal nocturnal hemoglobinuria — a well-known indication for Soliris.