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Synonyms: ACH-4471 | ACH4471
Compound class: Synthetic organic
Comment: Danicopan (ACH-4471) is an oral, direct inhibitor of the protease activity of complement factor D [1,4-5], which is a component of the alternative complement pathway. It was originally developed for potential to control complement-mediated intravascular hemolysis and prevent C3-mediated extravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria .
COVID-19: Danicoplan is being included in the ACTIV-5 platform trial NCT04988035 that is designed to identify promising agents as potential COVID-19 therapeutics that are suitable for progression to more definitive studies. It is initially being tested as a monotherapy and in combination with remdesivir in hospitalised COVID-19 patients.
Ligand Activity Visualisation Charts
These are box plot that provide a unique visualisation, summarising all the activity data for a ligand taken from ChEMBL and GtoPdb across multiple targets and species. Click on a plot to see the median, interquartile range, low and high data points. A value of zero indicates that no data are available. A separate chart is created for each target, and where possible the algorithm tries to merge ChEMBL and GtoPdb targets by matching them on name and UniProt accession, for each available species. However, please note that inconsistency in naming of targets may lead to data for the same target being reported across multiple charts.✖
|No information available.|
|Summary of Clinical Use|
|Danicopan is in clinical trial in patients with the complement-mediated disease paroxysmal nocturnal hemoglobinuria.|
|Clinical Trial ID||Title||Type||Source||Comment||References|
|NCT03053102||Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)||Phase 2 Interventional||Alexion Pharmaceuticals||3|
|NCT03472885||Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab||Phase 2 Interventional||Alexion Pharmaceuticals||2|
|NCT04988035||ACTIV-5 / Big Effect Trial (BET-C) for the Treatment of COVID-19||Phase 2 Interventional||National Institute of Allergy and Infectious Diseases (NIAID)|