Novartis' Iptacopan (Faberlin®) Gains New NMPA Approval for Full-Spectrum PNH Treatment in China

On May 29, Novartis China announced its oral monotherapyFeiheida®(Ipocapam Hydrochloride Capsules)Approved by NMPA for additional indications,For the treatment of paroxysmal nocturnal hemoglobinuria（PNH）Adult Patients. The expansion of this indication covers PNH patients who have already received complement inhibitor treatment, providing a new treatment option for this group.

As the world's first-of-its-kind specific complement B factor oral inhibitor,Ferahat® Approved for Three Indications in China, including adult PNH patients who have not previously received complement inhibitor treatment and adult C3 glomerulopathy（C3G）。

Source: Novartis Official WeChat

PNH is a chronic, progressive, life-threatening rare blood disease, with main clinical manifestations including intravascular hemolysis, bone marrow failure, and a high risk of thrombosis. Intravascular hemolysis leads to significant hemoglobin loss, causing anemia, which can result in ischemic and hypoxic damage to vital organs, leading to fatigue, muscle weakness, and decreased quality of life.

PNH is common in people aged 30-40, who are more eager to return to work and normal life. Among patients receiving anti-complement C5 therapy, 74% still experience persistent fatigue, and over one-third of patients require at least one blood transfusion per year.

This new indication approval is based on the head-to-head study results of APPLY-PNH.The study confirmed that PNH patients with residual anemia after prior treatment with anti-complement C5 therapy switched to treatment with ipocapant.Superior in hemoglobin level increase, FACIT-fatigue score improvement, and transfusion independence compared to continued anti-C5 complement therapy.。

APPLY-PNH Study Shows:

• PNH patients with residual anemia despite prior treatment with anti-complement C5 therapy, after switching to ipocapant treatment for 24 weeks,82.3% of patients had an increase in hemoglobin levels exceeding 2 g/dL, and 67.7% of patients achieved normalization of hemoglobin levels.（≥12 g/dL）, whereas the corresponding proportion among patients continuing with anti-complement C5 therapy was 0%.

• At the same time, Ipkaplan makes it possible to get rid of blood transfusions.95.2% of patients treated with Iptacopan avoided transfusions, whereas this proportion was only 45.7% in patients receiving anti-complement C5 therapy.

• In addition, the study also observed that Epcoritamab significantly improved fatigue, increasing the FACIT-Fatigue score to near normal.

Patients who complete the 24-week APPLY-PNH randomized treatment period may choose to enter the extension phase, continuing to receive ipocaplon or switching from anti-C5 to ipocaplon up to week 48. In the group continuously receiving ipocaplon, the outcomes achieved at week 24 were sustained through week 48.

In the group switching to ipilimumab after anti-complement C5 treatment, similar benefits were observed post-switch:

• Average hemoglobin levels increased to near-normal levels, and nearly all patients avoided transfusions.(94.1%, Weeks 26-48), after switching to Ipkapane, the FACIT-F fatigue score increased by 10.79 points, approaching normal.

• In terms of safety, Epcoritamab performs well.No new safety signals emerged within 48 weeks, and no treatment-related adverse events leading to treatment discontinuation occurred.（TEAEs）Clinical Breakthrough Hemolysis（BTH）The incidence of events was low, only 1/10 of that with anti-complement C5 therapy, and the severity was all mild to moderate.