This study aims to investigate how well the medication danicopan works when added to existing treatments ravulizumab or eculizumab. It focuses on children and teenagers who have a rare blood condition called paroxysmal nocturnal hemoglobinuria (PNH), which causes red blood cells to break down too easily, leading to a condition called extravascular hemolysis. The goal is to see if adding danicopan can better manage their condition by stabilizing their hemoglobin levels, which might help improve their overall health and reduce symptoms. Participants in this study will continue their current treatment with either ravulizumab or eculizumab and will also take danicopan. The effectiveness of this combination will be measured by checking changes in their hemoglobin levels after 12 weeks. By evaluating these changes, researchers hope to determine if danicopan provides additional benefits in managing the symptoms of PNH. The study will help doctors understand if this new treatment approach can offer better outcomes for young patients dealing with this challenging condition.
Inclusion Criteria: * Confirmed diagnosis of PNH. * CS-EVH defined by: Anemia: Hgb ≤ 11.0 g/dL, and absolute reticulocyte count ≥ 100 × 109/L * Treated with ravulizumab or eculizumab for at least 12 weeks immediately preceding Day 1, the dose received should be stable during this period, and there should be no anticipated changes in dosage or interval during the first 12 weeks of this study. * all participants must be vaccinated against meningococcal infection from serogroups A, C, W, and Y and serogroup B within 3 years prior to, or at least 14 days prior to Day 1 * vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae Exclusion Criteria: * Platelet count \< 30000/μL or there is a need for platelet transfusions. * ANC \< 500/μL. * Clinically significant laboratory abnormalities related to liver function, including: * ALT \> 2 × ULN or ALT \> 3 × ULN for participants with documented liver iron overload defined by serum ferritin values ≥ 500 ng/mL. * Direct bilirubin \> 2 × ULN, unless, in the Investigator's opinion, is due to hemolysis or Gilbert's syndrome based on medical history. * Current evidence of biliary cholestasis. * Known aplastic anemia or other bone marrow failure that requires HSCT or other therapies, including anti-thymocyte globulin and immunosuppressants unless the dosage of immunosuppressant has been stable for at least 12 weeks before Day 1 and is expected to remain stable through Week 12. * History of a major organ transplant (eg, heart, lung, kidney, liver) or HSCT. * Known or suspected complement deficiency. * Active bacterial or viral infection, a body temperature \> 38°C on 2 consecutive daily measures, evidence of other infection, or history of any febrile illness within 14 days prior to first study intervention administration.
is designated in this study