Novartis has announced positive results from the Phase IIIB APPULSE-PNH study, highlighting the effectiveness of Fabhalta (iptacopan), an oral monotherapy, in treating adult patients with paroxysmal nocturnal hemoglobinuria (PNH).
The trial showed that patients who transitioned from anti-C5 therapies, such as eculizumab or ravulizumab, experienced an improvement in average hemoglobin (Hb) levels after 24 weeks of treatment.
Overview of APPULSE-PNH Study
The APPULSE-PNH trial is a Phase IIIB, multicenter, single-arm, open-label study designed to assess the safety and efficacy of Fabhalta in PNH patients. Participants received 200 mg of the oral therapy twice daily for 24 weeks. The study enrolled 52 adults with stable treatment histories on anti-C5 therapies for at least six months before screening. These participants had baseline hemoglobin levels of 10 g/dL or higher and had not required red blood cell transfusions during that period.
The primary goal of the trial was to evaluate the change in hemoglobin levels after 24 weeks of treatment with Fabhalta. The results revealed a significant improvement compared to baseline, further solidifying the therapy’s potential to address unmet needs in PNH management.
Understanding Paroxysmal Nocturnal Hemoglobinuria
PNH is a rare and severe blood disorder caused by a genetic mutation in hematopoietic stem cells, which produce red and white blood cells and platelets. This mutation makes red blood cells vulnerable to destruction by the complement system, leading to both intravascular and extravascular hemolysis. The condition is associated with anemia, fatigue, blood clots, and other debilitating symptoms.
Patients with PNH are often treated with anti-C5 therapies, which target part of the complement system responsible for red blood cell destruction. However, for some patients, these therapies may not fully address the anemia or other symptoms, prompting the need for innovative treatments like Fabhalta.
Expanding Fabhalta’s Impact
The results from APPULSE-PNH build upon earlier findings from the Phase III program, which demonstrated the efficacy and safety of Fabhalta. The therapy has already received approval in the United States, European Union, Japan, and China for treating PNH, as well as accelerated approval in the U.S. for reducing proteinuria in adults with primary immunoglobulin A nephropathy (IgAN).
Novartis plans to present the full APPULSE-PNH data at a medical conference in 2025. This research further supports the potential of Fabhalta in addressing multiple complement-mediated diseases, offering new hope for patients with these challenging conditions.
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