New phase IIIB data shows Fabhalta (iptacopan) improved hemoglobin levels in adult patients with paroxysmal nocturnal hemoglobinuria who switched from anti-C5 therapy

After 24 weeks of treatment with Fabhalta, the average Hb level improved versus baseline.

“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,” said 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. “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.”

In the study, the safety profile of Fabhalta monotherapy was consistent with previously reported data.

Data will be presented at an upcoming medical meeting in 2025. Fabhalta was recently granted accelerated approval by the FDA  for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, and development is ongoing in multiple complement-mediated diseases.

About APPULSE-PNH; APPULSE-PNH (NCT05630001) is a Phase IIIB multicenter, single-arm, open-label study to evaluate the efficacy and safety of twice-daily oral Fabhalta (iptacopan) monotherapy (200mg) in adults with PNH who were switched from anti-C5 therapies (eculizumab or ravulizumab). The trial enrolled 52 participants who received Fabhalta for 24 weeks. Participants enrolled were required to be on a stable regimen with anti-C5 therapies (eculizumab or ravulizumab) for at least 6 months prior to screening with average hemoglobin (Hb) ≥10g/dL and no red blood cell transfusions in this period. The primary endpoint is change from baseline Hb levels after 24 weeks of treatment with Fabhalta.

PNH has a significant unmet need not addressed by anti-C5 therapies (eculizumab or ravulizumab). Anti-C5 therapies (eculizumab or ravulizumab) are commonly administered every 2-8 weeks as intravenous infusions, and treatment visits (including journey, waiting, infusion and recovery time) can take approximately 4 to 5 hours. Despite treatment with anti-C5 therapies, a large proportion of people with PNH remain anemic, and some dependent on blood transfusions.