Danicopan Regulatory Post-Marketing Study in Korea to Assess Safety and Effectiveness in Paroxysmal Nocturnal Hemoglobinuria

What this Trial Is About

Researchers are evaluating the safety and effectiveness of Danicopan as an add-on treatment to C5 inhibitors (Eculizumab or Ravulizumab) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) in Korea. This observational study aims to monitor known safety profiles and detect any unexpected adverse reactions in real-world clinical settings. It also seeks to assess how well Danicopan works when used alongside these existing therapies in routine medical practice. Participants in this study are patients prescribed Danicopan as an additional therapy to either Eculizumab or Ravulizumab under approved indications in Korea. The study focuses on treatment safety and effectiveness over a 12-week period. There is no experimental intervention assignment since this is an observational post-marketing study conducted under normal clinical conditions. During the study, safety data such as adverse events, serious adverse events, and drug reactions will be collected for up to 12 weeks from the first dose of Danicopan. Effectiveness will be evaluated through changes in hemoglobin levels, reticulocyte counts, fatigue scores, and the proportion of patients avoiding red blood cell transfusions at 12 weeks or at treatment end if earlier. Participants will receive routine clinical assessments without additional experimental procedures.

Danicopan PMS in Korea