*Study 301 was a randomized, open-label, active-controlled Phase 3 trial that evaluated the efficacy and safety of ULTOMIRIS® compared to eculizumab in adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who were clinically stable on eculizumab.4 Study 302 was a randomized, open-label, active-controlled Phase 3 trial assessing the efficacy and safety of ULTOMIRIS® in complement inhibitor-naïve adult patients with PNH.5
*N numbers include patients from Study 301 and Study 302.1,7
†Discontinued with AML, MDS, and lung adenocarcinoma. All were deemed unrelated to study drug.7
‡Causes of death were pulmonary sepsis, AML, lung adenocarcinoma, and malignant lung neoplasm. All were deemed unrelated to study drug.7
§The two deaths leading to study drug discontinuation were acute myeloid leukemia and lung adenocarcinoma.7
†Discontinued with AML, MDS, and lung adenocarcinoma. All were deemed unrelated to study drug.7
‡Causes of death were pulmonary sepsis, AML, lung adenocarcinoma, and malignant lung neoplasm. All were deemed unrelated to study drug.7
§The two deaths leading to study drug discontinuation were acute myeloid leukemia and lung adenocarcinoma.7
The most common adverse reactions (≥10%) with ULTOMIRIS® are headache, upper respiratory tract infection, nasopharyngitis, diarrhoea, pyrexia, nausea, arthralgia, back pain, fatigue, abdominal pain, dizziness and urinary tract infection.8
The most serious adverse reactions are meningococcal infection (0.7%) including meningococcal sepsis, meningococcal meningitis, encephalitis meningococcal, meningococcal infection and disseminated gonococcal infection (0.2%) including disseminated gonococcal infection and gonococcal infection.8
Please refer to the SmPC for further safety information.8
The most serious adverse reactions are meningococcal infection (0.7%) including meningococcal sepsis, meningococcal meningitis, encephalitis meningococcal, meningococcal infection and disseminated gonococcal infection (0.2%) including disseminated gonococcal infection and gonococcal infection.8
Please refer to the SmPC for further safety information.8
Among patients with PNH receiving ULTOMIRIS® or SOLIRIS® (eculizumab), the rate of survival did not differ from that of age and sex matched population control when those requiring treatment for bone marrow failure were excluded.†6,9
Figure adapted from Kulasekararaj A, et al. Ann Hematol. 2025;104(1):81-94.1
*Survival was not a prespecified endpoint of the 301 study or open-label extension, rather a post hoc analysis; the study was not powered to detect differences in survival. Death was a post hoc safety endpoint. ULTOMIRIS® data were compared with 414 untreated patients from the International PNH Registry. Survival probability was adjusted for age at PNH diagnosis, gender, and transfusion history.1,4
†Retrospective study comparing results from 389 patients with PNH, treated with SOLIRIS® and/or ULTOMIRIS®, between 2002 and 2022 vs. population mortality data taken from the human mortality database for the UK (1841–2020), stratified based on age and sex. The observed survival was not a prespecified endpoint, and the study was not powered to detect differences. This analysis excluded patients receiving an allogeneic HSCT, those with bone marrow dysfunction or those with clonal evolution to MDS or AML.9
ULTOMIRIS® Efficacy
Please refer to the ULTOMIRIS® Summary of Product Characteristics1 for further safety information.
Please report any adverse events via your national reporting system. Adverse events can also be reported to Alexion,
AstraZeneca Rare Disease by contacting: https://contactazmedical.astrazeneca.com/
AML, acute myeloid leukaemia; C5i, complement factor 5 inhibitor; CI, confidence interval; HSCT, haematopoietic stem cell transplant; IVH, intravascular haemolysis; MDS, myelodysplastic syndrome; PNH, paroxysmal nocturnal haemoglobinuria; SAE, serious adverse event; TEAE, treatment-emergent adverse event.