Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare blood disease where red blood cells break apart too easily. This study is testing a drug called iptacopan, to see if it's safe and works well over a long time. The study will give patients who finished earlier iptacopan studies a chance to continue using it. It is called open-label because both the researchers and participants know what treatment is being given. A single-arm study means all participants get the same treatment without comparing to another group.
- Participants must be 18 or older, with PNH, and have completed specific earlier studies.
- Prior vaccinations for certain infections are required.
- Study duration is long-term and no tapering down of medication is allowed.
To join, you should be healthy except for PNH and have no history of certain infections or stem cell transplants. The study aims to ensure iptacopan is safe and continues to be helpful for PNH patients.