2 July 2020 - - The European Commission has approved ULTOMIRIS (ravulizumab), a 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 who are complement inhibitor treatment-naïve or have received SOLIRIS (eculizumab) for at least three months and have evidence of response to eculizumab, US-based Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN) said.

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 % of adults and 29 % 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.

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 % improvement in serum creatinine from baseline).

In the initial 26-week treatment periods, 54 % of adults and 77.8% (interim data) of children demonstrated Complete TMA Response.

Treatment with ULTOMIRIS resulted in normalization of platelet count in 84% of adults and 94% of children, normalization of LDH (marker of hemolysis) in 77% of adults and 90% of children, and improved kidney function in 59 % of adults and 83 % (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, four 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% in adults and 94 % in children (interim data).

Treatment with ULTOMIRIS resulted in normalization of platelet count in 86 % of adults and 94% of children, normalization of LDH (marker of hemolysis) in 84 % of adults and 94% of children, and improved kidney function in 63% of adults and 94 % (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.