Novartis Plunges 6.14% on New PNH Treatment Approval

On May 29, 2025, Novartis experienced a significant drop of 6.14% in pre-market trading, reflecting investor concerns and market reactions to recent developments.

Novartis China announced that its oral monotherapy, Flynda (Iptacopan capsules), has received approval from the National Medical Products Administration (NMPA) for an additional indication to treat paroxysmal nocturnal hemoglobinuria (PNH) in adult patients. This expansion covers PNH patients who have previously received complement inhibitor therapy, providing them with a new treatment option. As the first global oral inhibitor of the complement B factor, Flynda's approved indications in China now include PNH patients who have not previously received complement inhibitor therapy and adult C3 glomerulopathy (C3G).

PNH is a rare, chronic, progressive, and life-threatening blood disorder characterized by intravascular hemolysis, bone marrow failure, and high-risk thromboembolism. The loss of large amounts of hemoglobin during intravascular hemolysis leads to anemia, which can cause ischemia and hypoxia in vital organs, resulting in fatigue, muscle weakness, and a decline in quality of life. PNH is commonly seen in individuals aged 30-40, who are eager to return to work and normal life. Among patients receiving anti-C5 therapy, 74% still experience fatigue, and more than one-third require blood transfusions at least once a year.

This new indication was approved based on the results of the APPLY-PNH head-to-head study, which demonstrated that PNH patients with residual anemia after anti-C5 therapy who switched to Iptacopan treatment showed improvements in hemoglobin levels, FACIT-fatigue scores, and reduced need for blood transfusions compared to those who continued anti-C5 therapy. The study found that 82.3% of patients who switched to Iptacopan had an increase in hemoglobin levels of more than 2 g/dL, and 67.7% achieved normal hemoglobin levels (≥12 g/dL), while none of the patients who continued anti-C5 therapy showed these improvements. Additionally, 95.2% of patients on Iptacopan avoided blood transfusions, compared to only 45.7% of those on anti-C5 therapy. Iptacopan also significantly improved fatigue, bringing FACIT-fatigue scores close to normal levels.