Novartis Announces Positive Results From PNH Study, Showing Improved Hb Levels With Fabhalta® In PNH Patients

Novartis announced today that the APPULSE-PNH Phase IIIB study of Fabhalta® (iptacopan) showed positive topline results. The study evaluated the efficacy and safety of twice-daily oral Fabhalta monotherapy in adults with paroxysmal nocturnal hemoglobinuria (PNH) who had switched from anti-C5 therapies, such as eculizumab or ravulizumab, and had a Hb level of ≥10g/dL following treatment. After 24 weeks of Fabhalta treatment, patients showed an improvement in average haemoglobin (Hb) levels compared to baseline.

Antonio Risitano, M.D., Ph.D., Chair of the International PNH Interest Group and Head of the Hematology and Hematopoietic Transplant Unit, Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria at the AORN San Giuseppe Moscati, Avellino, Italy, and APPULSE-PNH trial lead investigator, said in a statement, “These new results add to the body of evidence reinforcing that Fabhalta can benefit both patients previously treated with anti-C5 therapies studied in the APPULSE-PNH and APPLY-PNH trials and complement-inhibitor naïve patients studied in the APPOINT-PNH trial. Treatment goals for patients with PNH have greatly evolved, and we can now aim to resolve signs and symptoms of disease in most patients. It is promising to see this evolution, and we will continue to make progress to best support these patients.”

The safety profile of Fabhalta in this study was consistent with previously reported data. The results will be presented at a medical meeting in 2025. Fabhalta recently received accelerated approval from the US Food and Drug Administration (FDA) for reducing proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk for rapid disease progression, with ongoing development for other complement-mediated diseases.

David Soergel, M.D., Global Head, Cardiovascular, Renal and Metabolism Development Unit, Novartis, commented, “Across multiple clinical trials, Fabhalta has consistently shown clinically meaningful benefits for patients with PNH, and the APPULSE-PNH trial is a compelling addition to this body of evidence. These data reinforce our confidence in Fabhalta, the first and only oral monotherapy currently available for treating adults with PNH, to provide meaningful haemoglobin improvement, regardless of previous treatment experience.”