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, MA, USA I July 03, 2019 I 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.

About Paroxysmal Nocturnal Hemoglobinuria (PNH)
Paroxysmal nocturnal hemoglobinuria (PNH) is a chronic, progressive, debilitating and life-threatening ultra-rare blood disorder characterized by hemolysis (destruction of red blood cells) that is mediated by an uncontrolled activation of the complement system, a component of the body’s immune system.4,5,16 PNH can strike men and women of all races, backgrounds and ages without warning, with an average age of onset in the early 30s.4,10 PNH often goes unrecognized, with delays in diagnosis ranging from one to more than five years.11 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.6-9,12,13 The most devastating consequence of chronic hemolysis is thrombosis, which can occur in blood vessels throughout the body, damage vital organs and cause premature death.17 The first thrombotic event can be fatal.4,17,18 Despite historical supportive care, including transfusion and anticoagulation management, 20 to 35 percent of patients with PNH die within five to 10 years of diagnosis.19,20 Patients with certain types of hemolytic anemia, bone marrow disorders and unexplained venous or arterial thrombosis are at increased risk of PNH.11,21-25

About ULTOMIRIS®
ULTOMIRIS® (ravulizumab) is the first and only long-acting C5 inhibitor administered every eight weeks. It is approved in the EU 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. ULTOMIRIS (ravulizumab-cwvz) is approved in the U.S. and in Japan as a treatment for adults with PNH.

ULTOMIRIS 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 PNH, including atypical hemolytic uremic syndrome (aHUS), anti-acetylcholine receptor (AchR) antibody-positive myasthenia gravis (MG) and anti-aquaporin-4 (AQP4) auto-antibody-positive neuromyelitis optica spectrum disorder (NMOSD). In Phase 3 clinical studies in complement inhibitor-naïve patients with PNH14 and patients with PNH who had been stable on SOLIRIS® (eculizumab),15 intravenous treatment with ULTOMIRIS every eight weeks demonstrated non-inferiority to intravenous treatment with SOLIRIS every two weeks on all 11 endpoints.

The Phase 3 study of ULTOMIRIS, administered intravenously every eight weeks in adult patients with aHUS, met its primary objective. The U.S. Food and Drug Administration (FDA) has accepted for priority review a supplemental Biologics License Application (sBLA) for ULTOMIRIS for the treatment of people with aHUS in order to inhibit complement mediated thrombotic microangiopathy (TMA). Alexion plans to submit applications in the EU and Japan later in 2019. ULTOMIRIS is also currently being evaluated in a Phase 3 clinical study in children and adolescents with aHUS, administered intravenously every eight weeks. Alexion has initiated a Phase 3 study of ULTOMIRIS, intravenously administered every eight weeks, as a potential treatment for patients with generalized MG (gMG), and is planning to initiate a Phase 3 study in patients with NMOSD. In addition, Alexion has initiated Phase 3 studies of ULTOMIRIS delivered subcutaneously once per week as a potential treatment for patients with PNH, aHUS and gMG.

ULTOMIRIS has received Orphan Drug Designation (ODD) for the treatment of patients with PNH in the U.S. and Japan and for the subcutaneous treatment of patients with aHUS in the U.S.

About Alexion
Alexion is a global biopharmaceutical company focused on serving patients and families affected by rare diseases through the discovery, development and commercialization of life-changing therapies. As the global leader in complement biology and inhibition for more than 20 years, Alexion has developed and commercializes two approved complement inhibitors to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) as well as the first and only approved complement inhibitor to treat atypical hemolytic uremic syndrome (aHUS), anti-acetylcholine receptor (AchR) antibody-positive generalized myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD). Alexion also has two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare metabolic disorders, hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D). In addition, the company is developing several mid-to-late-stage therapies, including a second complement inhibitor, a copper-binding agent for Wilson disease and an anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G (IgG)-mediated diseases as well as several early-stage therapies, including one for light chain (AL) amyloidosis and a second anti-FcRn therapy. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on the core therapeutic areas of hematology, nephrology, neurology, and metabolic disorders. Alexion has been named to the Forbes’ list of the World’s Most Innovative Companies seven years in a row and is headquartered in Boston, Massachusetts’ Innovation District. The company also has offices around the globe and serves patients in more than 50 countries. This press release and further information about Alexion can be found at: www.alexion.com.

References
1. ULTOMIRIS Summary of Product Characteristics. Alexion Pharmaceuticals.
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14. Peffault de Latour R, Brodsky RA, Ortiz S et al. American Society of Hematology (ASH) Annual Meeting, San Diego, December 2, 2018;Session 101:2330.
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20. Loschi M, Porcher R, Barraco F, et al. Am J Hematol. 2016;91:366-370.
21. Borowitz MJ, Craig FE, DiGiuseppe JA, et al. Cytometry B Clin Cytom.2010;78B:211-230.
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* U.S. prescribing information included due to issuing country of the release

SOURCE: Alexion Pharmaceuticals

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