FDA approves Novartis’ Fabhalta for PNH treatment
Betsy Goodfellow | December 6, 2023 | News story | Medical Communications | FDA, Fabhalta, Haematology, Novartis, PNH
Novartis has announced that the US Food and Drug Administration has approved Fabhalta (iptacopan) as the first oral monotherapy for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH).
According to the company’s press release: “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]).”
The approval follows data from the phase 3 APPLY-PNH trial in patients with residual anemia despite prior anti-C5 treatment who have switched to Fabhalta. Throughout the clinical trials, the drug increased haemoglobin levels in the majority of patients, and in APPLY-PNH almost all patients treated with the drug did not require blood transfusions.
The most commonly reported adverse reactions included headache, nasopharyngitis, diarrhoea, abdominal pain, bacterial infection, nausea and viral infection.
Vinod Pullarkat, MD, MRCP, clinical professor, Department of Haematology and Haematopoietic Cell Transplantation, City of Hope, US, commented: “An efficacious oral treatment with a demonstrated safety profile could be practice-changing for physicians and help relieve burdens experienced by people with PNH. 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.”
Betsy Goodfellow
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