Novartis has announced that the US Food and Drug Administration (FDA) has given approval to Fabhalta (iptacopan) as the first oral monotherapy to treat adults suffering with paroxysmal nocturnal hemoglobinuria (PNH).
People suffering with PNH condition have an acquired mutation that makes their red blood cells susceptible to destruction in a premature way by the complement system.
A Factor B inhibitor, Fabhalta acts proximally in the alternative complement pathway of the immune system, offering thorough control of red blood cell (RBC) destruction within and outside the blood vessels (intra- and extravascular hemolysis [IVH and EVH]).
During clinical studies, treatment with Fabhalta raised hemoglobin levels (≥ 2 g/dL from baseline in the absence of RBC transfusions) in most patients. In APPLY-PNH study, it was found that almost all patients treated with Fabhalta did not get blood transfusions.
The approval from the FDA is based on the Phase III APPLY-PNH study in patients suffering with residual anemia (hemoglobin < 10 g/dL) despite previous anti-C5 treatment and who changed to Fabhalta. This medicine showed superiority in the improvement of hemoglobin despite no RBC transfusions as well as in transfusion avoidance rate over those patients who were put on anti-C5 treatments.
This clearance by the regulatory authority was also backed by the Phase III APPOINT-PNH study in complement inhibitor-naïve patients.
Novartis president for US Victor Bultó said: “The U.S. approval of Fabhalta is an extraordinary moment for people living with PNH, their loved ones and the healthcare providers who care for them.
“This new, effective oral medicine may mean that patients can reset their expectations of living with PNH, a chronic and life-altering blood disease. As Novartis continues to focus on conditions with unmet patient need, we are exploring the potential of Fabhalta in other complement-mediated diseases – with an ultimate goal to drive meaningful change for patients.”
Fabhalta was discovered and developed by Novartis, a pharma company whose medicines are claimed to reach more than 250 million people worldwide.
This medicine is expected to be available in the US from this month.