Overview
As part of a post-approval commitment, the Korean health authority requests a study to characterize safety and effectiveness in patients who are treated with Danicopan as an add-on to ravulizumab or eculizumab in normal clinical practice settings. This study is designed to assess the known safety profile or identify previously unsuspected adverse reactions and to evaluate the effectiveness of Danicopan under conditions of routine daily medical practice in Korea.
Description
The objectives of this study are to assess the safety and effectiveness of Danicopan in a real world setting in patients who are prescribed with the study drug under the approved indication in Korea.
Primary objective(s) To assess the safety of Danicopan as add-on therapy to a C5 inhibitor (Eculizumab or Ravulizumab)in patients with PNH in Korea.
Secondary objective(s) To assess effectiveness of Danicopan as add- on therapy to a C5 inhibitor (Eculizumab or Ravulizumab) in patients with PNH at 12 weeks.
Eligibility
Inclusion Criteria:
- Patients eligible for and treated with Danicpan as add-on therapy to a C5 inhibitor (Eculizumab or Ravulizumab) in patient with PNH in Korea
- Provision of a signed and dated written informed consent by the patient or their legally acceptable representative
Exclusion Criteria:
- Participation in any concurrent interventional trials during the period of study drug treatment
- Other off-label indications according to the approved label in Korea
