LONG-TERM SAFETY AND EFFICACY OF IPTACOPAN IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA: 4- AND 5-YEAR FOLLOW-UP OF PATIENTS FROM PHASE 2 STUDIES WHO ENTERED THE ROLL-OVER (SIE 2026) - Oct 6, 2026 - Abstract #B11; Pres time: Sep 22, 2026; 10:25 AM - 11:40 AM; Location: PLENARY HALL; P2, P3 | "BTH and MAVEs were rare and no new safety signals were observed. These results support iptacopan as a potentially practice-changing oral therapy for PNH 3."
Clinical • P2 data • Cardiovascular • Complement-mediated Rare Disorders • Hematological Disorders • Infectious Disease • Novel Coronavirus Disease • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases
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C. Frieri1; A. Risitano1,2,3; B. Ko4; L. Wong Lee Lee5; A. Röth6; F. Sicre-De-Fontbrune2,7; E. Soo Yap8; S. Yoon9; P. Ferber10; I. Baltcheva10; A. Verles10; S. Sanz11; L. Monaco10; T. Costigan10,12; R. Peffault De Latour2,7,13
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