Phase 3 data revealed that iptacopan, an oral, selective complement factor B inhibitor, significantly improves fatigue and quality of life among patients with paroxysmal nocturnal hemoglobinuria (PNH). The findings stem from two phase 3 trials, APPLY-PNH and APPOINT-PNH, which assessed changes from baseline to day 168 in fatigue and health-related quality of life during iptacopan treatment.
APPLY-PNH focused on iptacopan as a monotherapy in PNH patients with persistent anemia despite C5 inhibitor therapy, while APPOINT-PNH evaluated iptacopan in C5i-naïve PNH patients. Both trials demonstrated that iptacopan led to meaningful improvements in symptoms and impacts of PNH, as measured by the FACIT-Fatigue scale and EORTC QLQ-C30 subscales.
PNH, a rare disease characterized by hemolysis and thrombosis, often leads to anemia, fatigue, and other symptoms. Despite C5 inhibitor therapy, many patients experience persistent anemia and fatigue. Iptacopan's recent approval offers a new treatment avenue, showing efficacy and safety in improving fatigue and quality of life in both C5i-experienced and -naïve patients.
The trials also found correlations between improved FACIT-Fatigue scores and increased hemoglobin levels, as well as decreased lactate dehydrogenase levels, indicating clinical improvement in disease control. These results underscore iptacopan's potential to provide clinically meaningful disease control and enhance the quality of life for PNH patients after 168 days of treatment.
