Regis Peffault de Latour, MD, PhD, discusses the benefit observed with iptacopan vs standard-of-care eculizumab or ravulizumab in patients with paroxysmal nocturnal hemoglobinuria and residual anemia during the phase 3 APPLY-PNH trial.
Regis Peffault de Latour, MD, PhD, professor of hematology, Hematology and Bone Marrow Transplant Department of the Saint-Louis Hospital, Hôpital Saint Louis, in Paris, France, discusses the benefit observed with iptacopan vs standard-of-care eculizumab (Soliris) or ravulizumab (Ultomiris) in patients with paroxysmal nocturnal hemoglobinuria (PNH) and residual anemia during the phase 3 APPLY-PNH trial (NCT04558918).
Data from the study were presented during the 2022 ASH Annual Meeting. The trial met both co-primary end points for the percentage of patients with an increase in hemoglobin levels of at least 2 g/dL and at least 12 g/dL in the absence of red blood cell transfusions, Peffault de Latour says.
Fifty-one of 60 patients treated with iptacopan experienced a hemoglobin increase of at least 2 g/dL compared with 0 of 35 patients treated with SOC. Additionally, 42 of 60 patients in the iptacopan arm achieved a hemoglobin increase of at least 12 g/dL vs none in the SOC arm, Peffault de Latour concludes.