FDA Approves Fabhalta for the Treatment of Adults with Paroxysmal Nocturnal Hemoglobinuria (PNH)
Novartis today announced that the U.S. Food and Drug Administration has approved Fabhalta® (iptacopan) capsules as the first oral monotherapy for the treatment of adults with paroxysmal nocturnal hemoglobinuria (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 hemoglobin levels (≥ 2 g/dL from baseline in the absence of RBC transfusions) in a majority of patients and in APPLY-PNH nearly all patients treated with Fabhalta did not receive blood transfusions.
PNH has a significant unmet need not fully addressed by anti-C5 therapies (eculizumab or ravulizumab): despite treatment with anti-C5s, a large proportion of people with PNH may remain anemic, and dependent on blood transfusions.
It is estimated that approximately 10-20 people per million worldwide live with PNH. Although PNH can develop at any age, it is often diagnosed in people between 30-40 years old.
