Rare Daily Staff

The European Commission has granted marketing approval to Alexion Pharmaceuticals Ultomiris for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria, an ultra-rare and potentially deadly blood disorder that can lead to stroke and heart attack.

Paroxysmal nocturnal hemoglobinuria (PNH) is a chronic and progressive disease in which the complement system, part of the immune system, attacks a person’s own red blood cells. PNH can strike men and women of all races, backgrounds and ages without warning, with an average age of onset in the early 30s. Patients with PNH may experience a wide range of signs and symptoms, such as fatigue, difficulty swallowing, shortness of breath, abdominal pain, erectile dysfunction, dark-colored urine, and anemia. The condition can cause the formation of blood clots that can damage organs and cause premature death.

Ultomiris, a long-acting C5 complement inhibitor and the successor to the company’s Soliris, is administered every eight weeks for the treatment of adult patients with PNH who display clinical symptoms of high disease activity, and also for adult patients who are clinically stable after having been treated with Soliris for at least the past six months.

“More than a decade after Soliris was approved, Ultomiris provides a major step forward in the treatment of PNH,” said Alexander Röth, Department of Hematology, West German Cancer Center, University Hospital Essen, Germany. “Now PNH patients can experience greater freedom in their lives with significantly fewer infusions per year without any compromise on efficacy or safety. This has been impressively demonstrated in the largest phase 3 program conducted in PNH so far.”

The European Commission approval is based on comprehensive results from two phase 3 studies, which were recently published in Blood and represent the largest phase 3 program ever conducted in PNH.

In these studies, which included more than 440 patients who had either never been treated with a complement inhibitor before, or who had been stable on Soliris, the efficacy of Ultomiris administered every eight weeks was non-inferior to the efficacy of Soliris administered every two weeks on all primary and secondary endpoints.

The safety profile of Ultomiris was similar to that of Soliris. Additional data showed that Ultomiris provided immediate and complete C5 inhibition that was sustained for eight weeks between doses, and that Ultomiris eliminated breakthrough hemolysis associated with incomplete C5 inhibition. The entire clinical development program for Ultomiris in PNH to date represents more than 800 patient years of experience.

The U.S. Food and Drug Administration approved Ultomiris at the end 2018 for PNH and the Japanese Ministry of Health, Labour And Welfare followed in June with a similar approval for the drug.

“We believe Ultomiris will become the new standard of care for patients with PNH by providing immediate and complete C5 inhibition, sustained throughout the eight week dosing interval, and reducing the number of infusions per year from 26 with Soliris to only six or seven with Ultomiris,” said John Orloff, executive vice president and head of research and development at Alexion.

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

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