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December 06, 2023
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FDA approves Fabhalta for treatment of paroxysmal nocturnal hemoglobinuria

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The FDA approved iptacopan for treatment of adults with paroxysmal nocturnal hemoglobinuria.

Iptacopan (Fabhalta, Novartis) is an oral selective complement factor B inhibitor designed to be given as monotherapy for adults with paroxysmal nocturnal hemoglobinuria (PNH). The agent previously received orphan drug and breakthrough therapy designation for the indication from the FDA.

Image: Healio
FDA based approval of Fabhalta on results of the phase 3 APPLY-PNH trial.

“An efficacious oral treatment with a demonstrated safety profile could be practice-changing for physicians and help relieve burdens experienced by people with paroxysmal nocturnal hemoglobinuria,” Vinod Pullarkat, MD, MRCP, clinical professor in the department of hematology and hematopoietic cell transplantation at City of Hope, said in a Novartis-issued press release. “In clinical studies, iptacopan was superior to anti-C5s in hemoglobin improvement in the absence of [red blood cell] transfusion and transfusion avoidance rate, and also effective in complement inhibitor-naive individuals, by providing clinically meaningful hemoglobin-level increases without the need for blood transfusions.”

The FDA based its approval on results of the phase 3 APPLY-PNH trial for patients with PNH and residual anemia (hemoglobin < 10 g/dL) previously treated with an anti-C5 monoclonal antibody, and the phase 3 APPOINT-PNH study, for complement inhibitor-naive patients.

Patients who received prior anti-C5 therapies and switched to iptacopan had increased hemoglobin levels of 2 g/dL or more (82.3%) and 12 g/dL or more (67.7%) compared with those who did not receive iptacopan (0%). More than three-quarters of complement inhibitor-naive patients (77.5%) had increased hemoglobin levels greater than 2 g/dL after treatment with iptacopan.

Patients receiving iptacopan avoided transfusions at a significantly higher rate than those who received anti-C5 therapies (95.2% vs. 45.7%; P < .0001).

The most common adverse reactions for patients in the APPLY-PNH trial included headache, nasopharyngitis, diarrhea, abdominal pain, bacterial infection, nausea and viral infection. Serious reactions occurred in 3% of patients, including pyelonephritis, urinary tract infection and COVID-19.

Common adverse events for patients in the APPOINT-PNH study included headache, viral infection, nasopharyngitis and rash. Serious reactions occurred in 5% of patients, including COVID-19 and bacterial pneumonia.