Novartis factor B inhibitor wins first FDA nod for rare blood disorder

Dec. 7, 2023

The U.S. FDA has approved Novartis' Factor B inhibitor as the first oral monotherapy for the treatment of adults with the rare, chronic blood disorder, paroxysmal nocturnal hemoglobinuria (PNH).

People with PNH have an acquired mutation that makes red blood cells susceptible to premature destruction by the part of the immune system known as the complement system. The current standard of care, C5 inhibitor treatments administered as infusions, may leave symptoms, including hemolysis, bone marrow failure and thrombosis, uncontrolled.

According to Novartis, iptacopan, now branded Fabhalta, acts proximally in the alternative complement pathway of the immune system, providing comprehensive control of red blood cell destruction within and outside the blood vessels. In clinical trials, treatment with Fabhalta increased hemoglobin levels in the majority of patients.

While over one-third of patients on anti-C5 treatments require blood transfusions at least once per year, in Novartis' phase 3 APPLY-PNH trial, nearly all patients treated with Fabhalta did not receive blood transfusions.

Fabhalta met both primary endpoints in the open-label APPLY-PNH trial, proving superiority to AstraZeneca/Alexion’s currently marketed anti-C5 antibodies Soliris (eculizumab) and Ultomiris (ravulizumab) in adults with PNH.

Novartis hopes the nod in PNH is the first of many for the Factor B inhibitor, which is currently in development for a range of complement-mediated diseases including immunoglobulin A nephropathy (IgA nephropathy), C3 glomerulopathy (C3G), immune complex membranoproliferative glomerulonephritis (IC-MPGN) and atypical hemolytic uremic syndrome (aHUS).