ULTOMIRIS is the first and only long-acting C5 inhibitor administered every 8 weeks in adults. In maintenance dosing, ULTOMIRIS works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. ULTOMIRIS is indicated for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) and the treatment of adults and pediatric patients 1 month of age and older with atypical hemolytic uremic syndrome (aHUS).
ULTOMIRIS has received Orphan Drug Designation (ODD) for the treatment of patients with PNH in the United States and Japan.
ULTOMIRIS is approved in the United States and in Japan as a treatment for adults with PNH. ULTOMIRIS also is approved in the United States as a treatment for adults and pediatric patients 1 month of age and older with aHUS to inhibit complement-mediated thrombotic microangiopathy (TMA). The medication is approved in the European Union for the treatment of 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 SOLIRIS® (eculizumab) for at least the past 6 months. The European Commission also approved ULTOMIRIS 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 naive or have received eculizumab for at least three months and have evidence of response to eculizumab.
In the United States, ULTOMIRIS is available in two formulations. ULTOMIRIS 100 mg/mL is an advanced formulation of ULTOMIRIS 10 mg/mL that provides a quicker infusion time for PNH and aHUS patients. ULTOMIRIS 100 mg/mL has the same mechanism of action as ULTOMIRIS 10 mg/mL, with consistent safety and efficacy. Both formulations are administered via intravenous infusion.
ULTOMIRIS and aHUS
ULTOMIRIS is the first and only long-acting C5 inhibitor that provides immediate and complete inhibition that is approved for atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA).1
The phase 3 study of ULTOMIRIS, administered intravenously every eight weeks in adults and every four to eight weeks in pediatric patients one month of age and older with aHUS, met its primary endpoint of Complete TMA Response.
The safety and efficacy of ULTOMIRIS for the treatment of aHUS to inhibit complement-mediated TMA has been demonstrated in a global, multicenter, single arm, Phase 3 study which evaluated ULTOMIRIS administered by intravenous infusion in 56 adults (≥ 18 years of age) who were naïve to complement inhibitor treatment prior to study entry. The study consisted of an up to seven-day screening period, a 26-week initial evaluation period and an extension period of up to 4.5 years, which is still ongoing. The safety and efficacy of ULTOMIRIS is also being assessed in complement inhibitor-naïve children and adolescents (aged 18 and under) with a documented diagnosis of aHUS in a 26-week, ongoing, multicenter, single-arm, Phase 3 study.
The most frequently observed adverse reactions reported in this study were upper respiratory tract infection, diarrhea, nausea, vomiting, headache, hypertension and pyrexia.
ULTOMIRIS and PNH
ULTOMIRIS is a treatment option for adult patients living with paroxysmal nocturnal hemoglobinuria (PNH). ULTOMIRIS is the first and only long-acting C5 inhibitor that provides immediate and complete inhibition for 8 weeks.
In Phase 3 clinical studies in complement inhibitor-naïve patients with PNH2 and patients with PNH who had been stable on SOLIRIS,3 intravenous treatment with ULTOMIRIS every eight weeks demonstrated non-inferiority to intravenous treatment with SOLIRIS every two weeks on all 11 endpoints.