Clinical Research Directory

Key Inclusion Criteria * Male or female, aged 18 years and older * Diagnosed with PNH with clinically significant EVH as defined by anemia (Hgb ≤ 9.5 g/dL) with absolute reticulocyte count ≥ 120 × 109/L. * Currently receiving treatment with SOLIRIS® or ULTOMIRIS® for at least 6 months prior to initiation of danicopan treatment. * Platelet count ≥ 30,000/µL without the need for platelet transfusions. * Absolute neutrophil counts ≥ 500/µL. * Vaccinated against N meningitidis. Key Exclusion Criteria * Participants who are nursing or pregnant (or women who are planning to become pregnant during treatment with danicopan). * Laboratory abnormalities at screening, as follows: * Alanine aminotransferase (ALT) \> 2 × upper limit of normal (ULN) * Direct bilirubin \> 2 × ULN, with the exception of: * participants with increased bilirubin due to EVH in the opinion of the Treating Physician or * participants with documented Gilbert's syndrome * 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 initiating danicopan treatment.