These promising results presented at the EHA Annual Meeting underscore the potential for targeted Factor D inhibition with danicopan as an add-on to Ultomiris or Soliris to address clinically significant EVH while allowing patients to maintain disease control with established C5 complement inhibitors.”Īt the prespecified interim analysis for the ALPHA trial – occurring after 63 study participants either completed or discontinued from the primary treatment period of 12 weeks – danicopan met the primary efficacy endpoint. Gianluca Pirozzi, Senior Vice President, Head of Development, Regulatory and Safety, Alexion, said: “As a leader in PNH for decades, Alexion has transformed the treatment landscape by developing the first approved medicine for this rare disease and establishing C5 complement inhibition as standard of care. These results suggest danicopan has the potential to be an important option for the small subset of patients with PNH who experience clinically significant EVH while being treated with eculizumab or ravulizumab.” The ALPHA trial demonstrated that adding danicopan to standard of care with eculizumab or ravulizumab significantly improved fatigue and anaemia and reduced transfusion dependence, while still allowing for sustained control of IVH with terminal complement inhibition addressing the thrombotic risks associated with PNH. Mary’s Hospital of The Catholic University of Korea, and investigator in the ALPHA trial, said: “While EVH is not life-threatening, its manifestations can be burdensome for patients. Professor Jong Wook Lee, MD, PhD, Department of Haematology at Seoul St. 2-4 Approximately 10-20% of people living with PNH who are treated with a C5 inhibitor experience clinically significant EVH, which can result in continued symptoms of anaemia and require blood transfusions. PNH is a rare and severe blood disorder characterised by the destruction of red blood cells within blood vessels, known as intravascular haemolysis (IVH), and white blood cell and platelet activation that can cause thrombosis (blood clots) and result in organ damage and potentially premature death. The data were presented today at the European Hematology Association (EHA) Annual Meeting in Frankfurt, Germany. Positive results from the pivotal Phase III ALPHA trial showed that investigational, first-in-class oral Factor D inhibitor danicopan as add-on to standard of care C5 inhibitor therapy Ultomiris (ravulizumab) or Soliris (eculizumab) demonstrated a statistically significant and clinically meaningful increase in haemoglobin levels and maintained disease control in patients with paroxysmal nocturnal haemoglobinuria (PNH) who experience clinically significant extravascular haemolysis (EVH), compared to placebo plus established C5 inhibitor therapy.
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