Novartis Reports Positive Phase IIIB Results for Fabhalta in PNH

Novartis announced promising results from the Phase IIIB APPULSE-PNH trial of oral Fabhalta (iptacopan) in adults with paroxysmal nocturnal haemoglobinuria (PNH). The open-label study enrolled 52 patients with Hb ≥10g/dL who switched from anti-C5 therapies. After 24 weeks, patients saw a mean Hb increase of 2.01g/dL, with 92.7% reaching ≥12g/dL and none requiring transfusions.

Fatigue improved significantly, with FACIT-Fatigue scores matching the general population by day 168. The treatment also controlled intravascular and resolved extravascular haemolysis, shown by stable LDH and lower reticulocyte counts.

Novartis will present extended data from APPLY-PNH and APPOINT-PNH trials at the 2025 EHA Congress. Fabhalta, a Factor B inhibitor, represents a novel option for treating PNH. Additionally, the company shared positive interim results from the Phase III PSMAddition trial in PSMA-positive metastatic prostate cancer.

18-06-2025