This study is designed to understand how the drug **iptacopan** works and if it's safe for children with a condition called **paroxysmal nocturnal hemoglobinuria (PNH)**. PNH is a rare blood disease where red blood cells break down too early. The study will have children aged 2 to less than 18 years old participate over three separate groups based on age. It will last up to 60 weeks, with an 8-week screening, 26-week treatment, and 26-week extension period.

Participants must have confirmed PNH and meet specific health requirements. They should also be vaccinated against certain infections before starting the study. Children in this study can be currently receiving anti-C5 therapy, a treatment for blood issues, or they may be new to such treatments.

• The study involves a long commitment of up to 60 weeks.
• Vaccinations against certain infections are required before joining.
• A minimum of 12 children will be enrolled in this study.

It's important for potential participants and their guardians to understand the time commitment and health requirements involved in this study.