FDA approves Fabhalta as oral treatment for paroxysmal nocturnal hemoglobinuria

The US Food and Drug Administration has approved Fabhalta (iptacopan) as first oral monotherapy for adults suffering from paroxysmal nocturnal hemoglobinuria (PNH). PNH is a rare and chronic blood disorder where red blood cells are prone to premature destruction by the complement system. Despite prior anti-C5 treatments, a good number of patients with PNH still suffer with anemia, requiring blood transfusions.

The approval was based on the APPLY-PNH trial that revealed patients switching to Fabhalta experienced remarkable increases in hemoglobin levels, surpassing those continuing on anti-C5 treatment. Fabhalta demonstrated superior increases of hemoglobin levels ≥ 2 g/dL (82.3% vs. 0%) and hemoglobin level ≥ 12 g/dL (67.7% vs. 0%), both achieved without the need for red blood cell transfusions. This underscores the efficacy of Fabhalta in addressing the persistent unmet need for improved hemoglobin levels in PNH patients.

Fabhalta is a Factor B inhibitor targeting the complement pathway of immune system, crucial in complement-mediated hemolysis in PNH. The importance of this approval not only lies in its efficacy but also in being the first oral monotherapy for PNH, potentially offering a paradigm shift in the management of this rare blood disorder.

The approval comes after rigorous evaluation in the Phase III APPLY-PNH trial, where Fabhalta showcased its superiority in hemoglobin improvement in the absence of transfusions compared to anti-C5 treatments. Notably, patients avoiding transfusion reached an impressive rate of 95.2% with Fabhalta compared to 45.7% with anti-C5.

The most commonly reported adverse reactions with Fabhalta were headache, nasopharyngitis, diarrhea, abdominal pain, bacterial infection, nausea, and viral infection. Serious adverse reactions were reported in a small percentage of patients, including instances of pyelonephritis, urinary tract infection, COVID-19, and bacterial pneumonia.

Fabhalta is expected to be available in the US market soon, offering hope to those living with PNH and setting the stage for further exploration of its potential in other complement-mediated diseases. 

Source:

Novartis receives FDA approval for Fabhalta® (iptacopan), offering superior hemoglobin improvement in the absence of transfusions as the first oral monotherapy for adults with PNH. (2023, December 6). Novartis.