Dr Peffault de Latour on the Benefit of Iptacopan Vs SOC in PNH and Residual Anemia

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.