An open-label, single-arm, multicenter, phase 3 study to assess pharmacokinetics, safety and tolerability of iptacopan in pediatric PNH patients 2 to <18 years of age

Primary:
To evaluate the safety and tolerability of iptacopan during the 26-week treatment period.
To characterize the PK of iptacopan.

Secondary:
To evaluate the proportion of participants who achieve an increase in hemoglobin levels from baseline of ≥ 1 g/dL and the proportion of patients who achieve an increase in hemoglobin levels from baseline of ≥ 2 g/dL at 26 weeks and at 52 weeks (in the absence of RBC transfusions from Day 14 until the end of the 26-week and 52-week treatment periods).

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