ULTOMIRIS® (ravulizumab) Receives Marketing Authorization from European Commission for Adults with Paroxysmal Nocturnal Hemoglobinuria (PNH)

– ULTOMIRIS is the first approved, long-acting complement inhibitor for PNH, administered every other month, reducing the treatment burden for patients –

– ULTOMIRIS has the potential to become the new standard of care for both complement inhibitor-naïve patients and patients switching from SOLIRIS^® (eculizumab) –

BOSTON–(BUSINESS WIRE)–Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that the European Commission has approved ULTOMIRIS^® (ravulizumab) — the first and only long-acting C5 complement inhibitor administered every eight weeks — for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) with hemolysis with clinical symptoms indicative of high disease activity, and also for adult patients who are clinically stable after having been treated with SOLIRIS^® (eculizumab) for at least the past six months.¹

PNH is an ultra-rare and severe disease that, when left untreated, may cause a wide range of debilitating symptoms and complications, including thrombosis. Thrombosis occurs when a blood clot presents inside a blood vessel and slows or blocks the flow of blood through the circulatory system. Serious cases of thrombosis can occur throughout the body and result in organ damage, stroke, heart attack, and potentially premature death.^2-9

“More than a decade after SOLIRIS was approved, ULTOMIRIS provides a major step forward in the treatment of PNH,” said Dr. 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.”

PNH can strike men and women of all races, backgrounds and ages without warning, with an average age of onset in the early 30s.^3,10 PNH often goes unrecognized, with delays in diagnosis ranging from one to more than five years.¹¹

“At Alexion, our goal is to continue to improve the lives of people and families affected by PNH and other serious rare diseases,” said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. “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. We are also particularly pleased by the positive data showing patients can successfully transition from SOLIRIS to ULTOMIRIS.”

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.^1,12,13 In these studies, which included more than 440 patients who had either never been treated with a complement inhibitor before,¹⁴ or who had been stable on SOLIRIS,¹⁵ the efficacy of ULTOMIRIS administered every eight weeks was non-inferior to the efficacy of SOLIRIS administered every two weeks on all 11 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,¹⁴ and that ULTOMIRIS eliminated breakthrough hemolysis associated with incomplete C5 inhibition.¹⁵ 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 (FDA) approved ULTOMIRIS (ravulizumab-cwvz) for adults with PNH on December 21, 2018. The Japanese Ministry Of Health, Labour And Welfare (MHLW) approved ULTOMIRIS as a treatment for adults with PNH on June 18, 2019.