CHMP recommends approval of Ultomiris for atypical hemolytic uremic syndrome.- Alexion Pharma

Alexion Pharmaceuticals announced that the Committee for Medicinal Products for Human Use has adopted a positive opinion, recommending marketing authorization in the European Union for Ultomiris (ravulizumab) for the treatment of patients 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 3 months and have evidence of response to eculizumab.

The CHMP positive opinion 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. 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).

The most frequently observed adverse reactions reported in these studies were upper respiratory tract infection, diarrhea, nausea, vomiting, headache, hypertension and pyrexia.