Novartis Fabhalta gets positive CHMP opinion as first oral monotherapy for adult patients with paroxysmal nocturnal hemoglobinuria

The positive CHMP decision is based on robust data from the Phase III APPLY-PNH study in patients with residual anemia despite prior anti-C5 treatment who switched to Fabhalta vs. patients who stayed on anti-C5 treatment, and the Phase III APPOINT-PNH study in complement-inhibitor naïve patients. In APPLY-PNH, at 24 weeks, 82.3% of anti-C5-experienced Fabhalta patients achieved a sustained increase of Hb levels ≥2 g/dL from baseline in the absence of transfusions vs. 2.0% for anti-C5 (difference of 80.2%, P<0.0001); in APPOINT-PNH, 92.2% of complement inhibitor-naïve patients using Fabhalta achieved this outcome. Similarly, in APPLY-PNH, results highlighted a transfusion avoidance rate of 94.8% for anti-C5-experienced Fabhalta patients vs. 25.9% for those on anti-C5 (difference of 68.9%, P<0.0001). In both studies, Fabhalta was also shown to control the destruction of red blood cells (RBCs) within the blood vessels, known as intravascular hemolysis (IVH), with mean lactate dehydrogenase (LDH) levels maintained at <1.5 x upper limit of normal. In APPLY-PNH, patients reported improvements in fatigue as measured by Functional Assessment of Chronic Illness Therapy – Fatigue [FACIT-F] scores. The safety profile of iptacopan was consistent across both APPLY-PNH and APPOINT-PNH studies.

“If approved by the European Commission, Fabhalta would be the first oral monotherapy available to PNH patients in Europe,” said Patrick Horber M.D., President, International, Novartis, Basel. “With current standard of care, PNH symptoms are often uncontrolled, while patients endure regular and time-consuming infusions. This oral therapy could provide a much-needed alternative to support many people living with PNH who often have to structure their lives around managing their condition.’’

Following the CHMP’s recommendation to approve Fabhalta in adult patients with PNH who have hemolytic anemia, the European Commission (EC) will take a final decision within approximately two months.