(222) Real-world iptacopan use among United States patients with paroxysmal nocturnal hemoglobinuria

Background: : Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening hematological disorder affecting ≈24/1,000,000 people in the US. Iptacopan, the first orally administered monotherapy for PNH, received US Food and Drug Administration approval in December 2023. APPRISE-PNH is a data platform designed to capture comprehensive real-world information on iptacopan-treated patients with PNH.

Objective: : To describe patient profiles and assess treatment patterns and adherence among iptacopan-treated patients (age ≥18 years) with PNH in the US real-world setting.

Methods: : The present interim analysis of APPRISE-PNH includes data from patients enrolled from 7/2/2024-5/2/2025, with additional data collection expected through 2025. Duration of therapy was measured from the date of iptacopan initiation until discontinuation or data cut-off, whichever occurred first. Treatment adherence was measured using the medication possession ratio (MPR; ratio of the total days’ supply to days covered) and the proportion of days covered (PDC; ratio of days with supply to days covered).

Results: : Among 47 patients enrolled as of May 2, 2025, median age was 50.0 years, 59.6% were female, and 57.4% were covered by commercial insurance. Overall, 27.7% of patients (n=13) were treatment naive at the time of iptacopan initiation, while 72.3% (n=34) had received prior complement inhibitor therapy for PNH. The median (IQR) duration of iptacopan therapy was 282.0 days (120.5–364.5). At the time of data cut-off, 2 patients discontinued therapy (1 provider decision, 1 death). All patients received iptacopan 200 mg twice daily (as per the product label). The median (IQR) MPR and PDC were 105% (101%–111%) and 98% (94%–100%), respectively.

Conclusions: : These data represent early insights into the real-world use of iptacopan in US patients with PNH. Adherence to iptacopan was high, as indicated by the MPR and PDC. Enrollment is currently ongoing; data on additional patients, laboratory and clinical outcomes, and longer follow-up are forthcoming.

Sponsorship:: Novartis Pharmaceuticals Corporation