EU approves Ultomiris for patients with atypical hemolytic uremic syndrome.- Alexion Pharma

Alexion Pharmaceuticals 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 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 a minimum of 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.