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Alexion Pharmaceuticals Announces ULTOMIRIS Receives EC Marketing Authorization for Adults and Children with Atypical Hemolytic Uremic Syndrome (aHUS)

Alexion Pharmaceuticals, Inc.

Benzinga · -
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 adults and children with a body weight of 10 kg or above with atypical hemolytic uremic syndrome (aHUS) who are complement inhibitor treatment-naïve or have received SOLIRIS® (eculizumab) for at least three months and have evidence of response to eculizumab. "The consequences of aHUS are severe and potentially life-threatening, creating significant challenges and uncertainty for patients and their families. The goal of aHUS treatment is to prevent the body from attacking itself through the inhibition of uncontrolled C5 complement activation – a part of the body's immune system," said Prof. Dr. Hermann Haller, Clinic for Nephrology, University Hannover, Germany. "Clinical study results showed that both adults and children with aHUS had immediate and complete C5 inhibition following the first dose of ULTOMIRIS sustained for up to eight weeks. In addition to having demonstrated clinically meaningful benefits in people with aHUS, ULTOMIRIS provides greater freedom with significantly fewer infusions per year." Atypical HUS is an ultra-rare disease that can cause progressive injury to vital organs, primarily the kidneys, via damage to the walls of blood vessels and blood clots. Atypical HUS affects both adults and children and many patients present in critical condition in the hospital setting, often requiring supportive care, including dialysis, in an intensive care unit. The prognosis of aHUS can be poor in many cases, with 56 percent of adults and 29 percent of children developing end-stage renal disease or dying within a year of diagnosis with supportive care alone,3 so a timely and accurate diagnosis – in addition to treatment – is critical to improving patient outcomes. "At Alexion, our goal is to continue to improve the lives of people and families affected by aHUS and other serious rare diseases," said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. "Treatment with ULTOMIRIS offers convenient eight-week dosing, which we believe is preferred by patients because it provides them with greater flexibility and improved quality of life, while also reducing the burden on healthcare systems that currently face significant strain in many countries. Today's marketing authorization marks an important step in our efforts to establish ULTOMIRIS as the new standard of care for the aHUS patient community." The European Commission approval is based on data from two global, single-arm open-label studies of ULTOMIRIS – one in adults and one in children, referred to as pediatrics in the study – with aHUS. Both studies are ongoing. A total of 18 out of 21 complement inhibitor treatment-naïve children and 56 out of 58 complement inhibitor treatment-naïve adults were enrolled and included in the interim analysis. Efficacy evaluation of Complete TMA Response was defined by normalization of hematologic parameters (platelet count and LDH) and improved kidney function (as measured by ≥25 percent improvement in serum creatinine from baseline). In the initial 26-week treatment periods, 54 percent of adults and 77.8 percent (interim data) of children demonstrated Complete TMA Response. Treatment with ULTOMIRIS resulted in normalization of platelet count in 84 percent of adults and 94 percent of children, normalization of LDH (marker of hemolysis) in 77 percent of adults and 90 percent of children, and improved kidney function in 59 percent of adults and 83 percent (interim data) of children (for patients on dialysis at enrollment, baseline was established after they had come off dialysis). In the 52-week follow-up period, 4 additional adult patients and 3 pediatric patients had a Complete TMA Response that was confirmed after the 26-week Initial Evaluation Period resulting in an overall Complete TMA Response of 61 percent in adults and 94 percent in children (interim data). Treatment with ULTOMIRIS resulted in normalization of platelet count in 86 percent of adults and 94 percent of children, normalization of LDH (marker of hemolysis) in 84 percent of adults and 94 percent of children, and improved kidney function in 63 percent of adults and 94 percent (interim data) of children (for patients on dialysis at enrollment, baseline was established after they had come off dialysis). A second cohort of 10 pediatric patients who were SOLIRIS-experienced were included in the pediatric study, demonstrating that switching to ULTOMIRIS maintained disease control as evidenced by stable hematologic and renal parameters, with no apparent impact on safety. The most frequently observed adverse reactions reported in these studies were upper respiratory tract infection, diarrhea, nausea, fatigue, headache, nasopharyngitis, and pyrexia. Serious meningococcal infections have occurred in patients treated with ULTOMIRIS, however in aHUS studies, no meningococcal infections occurred in the 89 patients receiving treatment with ULTOMIRIS. To minimize the risk for patients, specific risk-mitigation plans, including a Risk Management Plan, have been established for ULTOMIRIS. The U.S. Food and Drug Administration (FDA) approved ULTOMIRIS for the treatment of aHUS to inhibit TMA for adult and pediatric (one month of age and older) patients in October 2019. A regulatory filing for marketing authorization of ULTOMIRIS for the treatment of aHUS in Japan is currently under review. ULTOMIRIS is also approved for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) in the U.S and Japan, and in the EU as a treatment for adult patients with 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 eculizumab for at least the past six months.