PHARMA NEWS | FDA Approves Iptacopan for Paroxysmal Nocturnal Hemoglobinuria

Novartis announced that the U.S. Food and Drug Administration (FDA) approved Fabhalta®(iptacopan) as the first oral monotherapy for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH).

Fabhalta is a Factor B inhibitor that acts proximally in the alternative complement pathway of the immune system, providing comprehensive control of red blood cell (RBC) destruction within and outside the blood vessels (intra- and extravascular hemolysis [IVH and EVH]). In clinical trials, treatment with Fabhalta increased haemoglobin levels (≥ 2 g/dL from baseline in the absence of RBC transfusions) in the majority of patients and in APPLY-PNH nearly all patients treated with Fabhalta did not receive blood transfusions.

Data from the 24-week core treatment periods in both trials showed that patients experienced a sustained increase of hemoglobin levels of at least 2 g/dL from baseline without red blood cell (RBC) transfusions. Specifically, 82.3% of patients who had prior exposure to anti-C5 agents and received iptacopan responded vs 0% of those who continued on anti-C5 agents (difference, 81.5%; < .0001). Moreover, 77.5% of patients who were naive to complement inhibitors achieved this outcome; on sensitivity analysis, this rate was 87.5%.

Moreover, 67.7% of patients who had prior exposure to an anti-C5 agent and received iptacopan responded compared with 0% of those on continued treatment with anti-C5 agents (difference, 66.6%; < .0001). Those with prior exposure to anti-C5 agents who received iptacopan achieved an RBC transfusion avoidance rate of 95.2% vs 45.7% for those on continued treatment with anti-C5 agents (difference, 49.5%; < .0001).

“An efficacious oral treatment with a demonstrated safety profile could be practice-changing for physicians and help relieve burdens experienced by people with PNH,” Vinod Pullarkat, MD, MRCP, clinical professor in the Department of Hematology and Hematopoietic Cell Transplantation at City of Hope, stated in a press release. “In clinical studies, iptacopan was superior to anti-C5s in hemoglobin improvement in the absence of RBC transfusion and transfusion avoidance rate, and also effective in complement inhibitor-naïve individuals, by providing clinically meaningful hemoglobin-level increases without the need for blood transfusions.”

Source: Novartis Press Release

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