AstraZeneca will present clinical and real-world data on multiple hematological conditions and rare diseases, including paroxysmal nocturnal hemoglobinuria (PNH), at the European Hematology Association (EHA) 2023 Hybrid Congress, June 8-11, 2023.

One of the poster presentations will disclose data from the pivotal ALPHA phase 3 trial evaluating the danicopan (ALXN2040), a investigational first-in-class oral factor D inhibitor, as an add-on to standard of care C5 inhibitor therapy ravulizumab (Ultomiris®) or eculizumab (Soliris®).

According to a prespecified interim efficacy, danicopan has the potential to improve hemoglobin levels and reduce the need for transfusions in patients with PNH who experience clinically significant extravascular hemolysis while on standard of care.

The prevalence of clinically significant extravascular hemolysis in patients with PNH who are receiving C5 inhibitor therapy ranges from 7% to 21%, according to real-world and clinical trial data that will be presented as a conference abstract.

Read more about PNH therapies

“We look forward to presenting results at EHA from the ALPHA Phase III trial showing the potential for danicopan, as an add-on to Ultomiris or Soliris, to address clinically significant EVH, which can impact approximately 10% to 20% of PNH patients treated with C5 inhibitors, while allowing them to maintain treatment with standard of care. Additional presentations will highlight our momentum in expanding our pipeline beyond complement to address the needs of more people living with rare diseases,” said Gianluca Pirozzi, senior vice president, head of development, regulatory and safety at Alexion Pharmaceuticals, the group within AstraZeneca focused on rare diseases.

In addition, AstraZeneca will present the following data on PNH:

  • An analysis of outcomes up to 4 years from the pivotal, phase 3 trial and open-label extension of ravulizumab in C5 inhibitor-experienced adults with PNH:
    • The results support the efficacy of ravulizumab in controlling intravascular hemolysis while maintaining favorable safety and tolerability profile. The survival rate throughout the 4-year study period was 98.4%.
  • An analysis of Korean registry data in patients living with PNH:
    • The results demonstrate that the levels of lactate dehydrogenase, but not hemoglobin levels, can predict thromboembolism, which, in turn, predicts death.
  • Insights into disease control and treatment satisfaction among patients and physicians.


AstraZeneca showcases scientific advances across rare and malignant haematological conditions at EHA 2023. News release. AstraZeneca; May 16, 2023.