By Fineline Cube 
Swiss pharmaceutical major Novartis (NYSE: NVS) has released positive topline results from the APPULSE-PNH Phase IIIb study for its complement inhibitor Fabhalta (iptacopan). This study marks a significant milestone in the evaluation of Fabhalta’s efficacy and safety in treating adult patients with paroxysmal nocturnal hemoglobinuria (PNH).
APPULSE-PNH Study Design and Outcomes
The APPULSE-PNH study is designed to assess the efficacy and safety of twice-daily oral monotherapy Fabhalta in adult patients with PNH who were switched from anti-C5 therapies, provided they have a hemoglobin (Hb) level of ≥10g/dL following treatment with eculizumab or ravulizumab. The latest data indicates that Fabhalta can effectively improve the average Hb levels in adult patients with PNH treated with anti-C5 therapy, maintaining a safety profile consistent with previous findings.
Understanding PNH and the Role of Fabhalta
PNH is a rare, chronic, and serious complement-mediated blood disorder characterized by hemolysis, anemia, thrombosis, and other symptoms. Patients with PNH are often treated with anti-C5 therapies. Fabhalta, a first-in-class specific oral complement Factor B inhibitor, offers a new approach to managing this condition. Its approval in China in April this year for use in adult patients with PNH who were previously untreated with complement inhibitors highlights the potential impact of this treatment on the global PNH patient population.-Fineline Info & Tech