Rare Daily Staff

The U.S. Food and Drug Administration granted priority review to Alexion Pharmaceuticals’ application to expand the approved use of its long-acting C5 complement inhibitor Ultomiris to include the treatment of people with atypical hemolytic uremic syndrome.

Atypical HUS (aHUS) is a severe and chronic ultra-rare disease that can cause progressive damage to vital organs, predominantly the kidneys, leading to kidney failure and premature death. Alexion is seeking approval to expand the indication of Ultomiris to include aHUS in order to inhibit complement-mediated thrombotic microangiopathy. The FDA has set a target action date of October 19, 2019 on the application.

Ultomiris, which is already approved to treat paroxysmal nocturnal hemoglobinuria, is a monoclonal antibody that works to quiet the immune response. It works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. The terminal complement cascade, when activated in an uncontrolled manner, plays a role in severe ultra-rare disorders like aHUS.

“This acceptance is an important step in our efforts to deliver a potential new standard of care to people living with this devastating disease,” said John Orloff, executive vice president and head of research and development at Alexion.

Alexion’s application is based on previously announced results, which were recently presented at the European Renal Association – European Dialysis and Transplant Association Congress, from the phase 3 study of Ultomiris in people with aHUS, which met the primary endpoint of complete TMA response, defined by hematologic normalization and improved kidney function.

Photo: John Orloff, executive vice president and head of research and development at Alexion

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