The efficacy and safety of ULTOMIRIS® were evaluated in two pivotal Phase 3 trials.1,2
The efficacy and safety of Ultomiris® was assessed in a multicentre, single-arm, Phase 3 study conducted in paediatric patients. The primary efficacy endpoint was complete TMA response, defined as platelet count normalisation (≥150 × 109/L), LDH normalisation (≤246 U/L), and ≥25% improvement in SCr from baseline.1‡
The study consisted of a 26-week initial evaluation period and patients were allowed to enter an extension period of up to 4.5 years. A total of 21 complement inhibitor treatment-naïve children with evidence of TMA* were enrolled, of which 18 were included in the full analysis set.1
The study consisted of a 26-week initial evaluation period and patients were allowed to enter an extension period of up to 4.5 years. A total of 21 complement inhibitor treatment-naïve children with evidence of TMA* were enrolled, of which 18 were included in the full analysis set.1
‡Patients had to meet each complete TMA response criteria at two separate assessments obtained at least 28 days apart and any measurement in between.1
*Evidence of active TMA (hemolysis, thrombocytopenia, and kidney dysfunction) per the following laboratory criteria: platelet count, <150 x 109 /L; LDH, ≥1.5 x upper limit of normal; hemoglobin, ≤ lower limit of normal; and serum creatinine level ≥ 97 .5th percentile for age.1
†Patients who receive a meningococcal vaccine less than 2 weeks before initiating ravulizumab treatment must receive treatment with appropriate prophylactic antibiotics until 2 weeks after vaccination. Patients who have not been vaccinated prior to initiating ravulizumab treatment should receive prophylactic antibiotics prior to and for at least 2 weeks after meningococcal vaccination.1
†Patients who receive a meningococcal vaccine less than 2 weeks before initiating ravulizumab treatment must receive treatment with appropriate prophylactic antibiotics until 2 weeks after vaccination. Patients who have not been vaccinated prior to initiating ravulizumab treatment should receive prophylactic antibiotics prior to and for at least 2 weeks after meningococcal vaccination.1
Complete TMA response as defined by:
Adverse Event Reporting
Please report any adverse events via your national reporting system. Adverse events can also be reported to Alexion Pharmaceuticals by contacting: https://contactazmedical.astrazeneca.com/
eGFR, estimated glomerular filtration rate; ICU, intensive care unit; LDH, lactate dehydrogenase; TMA, thrombotic microangiopathy.
The efficacy and safety of ULTOMIRIS® in patients with aHUS was assessed in a multicentre, single-arm Phase 3 study of adult patients who displayed signs of active TMA.1 A total of 56 patients who were naïve to C5 complement inhibitor treatment prior to study entry were evaluated for efficacy. The study consisted of a 26-week initial evaluation period and patients were allowed to enter an extension period for up to 4.5 years.3
The primary efficacy endpoint was complete TMA response, defined as platelet count normalisation (≥150 × 109/L), LDH normalisation (≤246 U/L), and ≥25% improvement in SCr from baseline.1‡
The primary efficacy endpoint was complete TMA response, defined as platelet count normalisation (≥150 × 109/L), LDH normalisation (≤246 U/L), and ≥25% improvement in SCr from baseline.1‡
‡Patients had to meet each complete TMA response criteria at two separate assessments obtained at least 28 days apart and any measurement in between.1
*Evidence of active TMA (hemolysis, thrombocytopenia, and kidney dysfunction) per the following laboratory criteria: platelet count, <150 x 109 /L; LDH, ≥ 1.5 x upper limit of normal; serum creatinine level ≥ upper limit of normal.1
†Patients who received meningococcal vaccine less than 2 weeks before initiating ravulizumab treatment must have received treatment with appropriate prophylactic antibiotics until 2 weeks after vaccination. Patients who had not been vaccinated prior to initiating ravulizumab treatment should have received prophylactic antibiotics prior to and for at least 2 weeks after meningococcal vaccination.1
†Patients who received meningococcal vaccine less than 2 weeks before initiating ravulizumab treatment must have received treatment with appropriate prophylactic antibiotics until 2 weeks after vaccination. Patients who had not been vaccinated prior to initiating ravulizumab treatment should have received prophylactic antibiotics prior to and for at least 2 weeks after meningococcal vaccination.1
§Dialysis at baseline was defined as within 5 days of first dose.
Complete TMA response as defined by:
Adverse Event Reporting
Please report any adverse events via your national reporting system. Adverse events can also be reported to Alexion Pharmaceuticals by contacting: https://contactazmedical.astrazeneca.com/
eGFR, estimated glomerular filtration rate; ICU, intensive care unit; LDH, lactate dehydrogenase; TMA, thrombotic microangiopathy; TTP, thrombotic thrombocytopenic purpura.