Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab

Key Inclusion Criteria:

Diagnosed with PNH
Have received at least one red blood cell transfusion within last 12 weeks
Anemia with adequate reticulocytosis
Must be on a stable regimen of eculizumab
Platelet count ≥ 40,000/microliter without the need for platelet transfusions
Documentation of vaccination for Neisseria meningitidis, Haemophilus influenza, and Streptococcus pneumoniae or willingness to receive vaccinations based on local guidelines
Willingness to receive antibiotic prophylaxis
Female participants must use highly effective birth control to prevent pregnancy during the clinical trial and for 30 days after their last dose of study drug
Male participants must use a highly effective birth control with a female partner to prevent pregnancy during the clinical trial and for 90 days after the last dose of study drug

Key Exclusion Criteria:

Current evidence of bone marrow failure or aplastic anemia requiring treatment
History of a major organ transplant or hematopoietic stem cell/marrow transplant
Received another investigational agent within 30 days or 5 half-lives of the investigational agent prior to study entry, whichever is greater
Documented C5 complement protein mutations
Known or suspected complement deficiency
Contraindication to any of the required vaccinations
Active bacterial infection or clinically significant active viral infection, a body temperature >38°C, or other evidence of infection
History of meningococcal infection, or a first-degree relative or household contact with a history of meningococcal infection
History of hypersensitivity reactions to commonly used antibacterial agents

Note: Additional inclusion/exclusion criteria may apply, per protocol.