IPTACOPAN ASH UPDATE
Significant unmet need remains in PNH despite current
standard of care anti-C5 therapy
PNH prevalence 10-20 cases/million = ~6k patients in the US1
63%
of patients treated with
a terminal complement
inhibitor showed signs
of ongoing hemolysis²
~1/3
of patients reported having
≥1 RBC transfusion despite
treatment with terminal
complement inhibitors 3,4
>75%
of patients treated with
terminal complement inhibitors
reported fatigue symptoms³
1. Cançado RD, 2021 and Jalbert JJ, 2019, Mon Pere N, 2018. 2. Risitano AM et al. Blood. 2009;113(17):4094-4100. 3. Dingli D et al. Ann Hematol. 2022;101(2):251-263.
4. Kulasekararaj AG et al. Eur J Haematol. 2022;109(3):205-214. doi:10.1111/ejh.13783.
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IPTACOPAN ASH UPDATE | DECEMBER 13, 2022
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