Novartis has reported positive outcomes from the multi-centre Phase IIIB APPULSE-PNH study of oral Fabhalta (iptacopan) in adults with paroxysmal nocturnal haemoglobinuria (PNH).
These findings are set to be presented at the European Hematology Association (EHA) Congress in 2025.
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The single-arm, open-label, multinational trial assessed the oral therapy in PNH subjects with haemoglobin (Hb) levels of ≥10g/dL, who transitioned from anti-C5 therapies (eculizumab or ravulizumab).
It enrolled 52 subjects and aimed to expand the clinical evidence for the therapy by including patients with higher baseline Hb levels than those in the pivotal Phase III programme.
Following 24 weeks of treatment with the therapy, subjects experienced an average increase in Hb levels of 2.01g/dL, with a significant number achieving normal or near-normal levels.
During the trial, none of the participants needed blood transfusions, and 92.7% of them reached Hb levels of at least 12g/dL.
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By GlobalDataIn addition, subjects reported improvements in fatigue, with Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scores aligning with those of the general population by day 168.
Novartis noted that Fabhalta-treated subjects also maintained intravascular haemolysis control and resolved extravascular haemolysis control, indicated by lactate dehydrogenase levels remaining below 1.5 times the upper limit of normal and a decrease in absolute reticulocyte count.
Along with these findings, the company will also present longer-term data from the APPLY-PNH and APPOINT-PNH Phase III trials at the EHA Congress.
Novartis development president and chief medical officer Shreeram Aradhye said: “New data from APPULSE-PNH, combined with findings from the Phase III roll-over extension of the APPLY-PNH and APPOINT-PNH studies, reinforce the efficacy and safety profile of Fabhalta in delivering real benefits to patients.”
Fabhalta was discovered by Novartis and acts as a Factor B inhibitor of the alternative complement pathway. It also offers a new treatment option for the rare and life-threatening complement-mediated blood disorder, PNH.
The company recently shared topline outcomes from the Phase III PSMAddition trial’s pre-specified interim analysis, in which Pluvicto plus standard of care (SoC) treatment demonstrated a benefit in treating prostate-specific membrane antigen (PSMA)-positive metastatic hormone-sensitive prostate cancer (mHSPC).
