Danicopan (Voydeya®) is an oral complement factor D inhibitor developed by Alexion AstraZeneca Rare Disease as an add-on treatment to ravulizumab or eculizumab for patients with clinically significant extravascular hemolysis. It received its first approval in Japan on 18 January 2024 for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) when used in combination with a complement component 5 (C5) inhibitor. The European Medicines Agency (EMA) also adopted a positive opinion recommending marketing authorization for danicopan for PNH patients who still experience residual hemolytic anemia despite treatment with a C5 inhibitor. This summary outlines the key milestones in danicopan's development leading to its first approval for PNH. The information presented here reflects the author's opinions. For a complete list of declarations, including funding and author disclosure statements, and copyright information, please refer to the full text online. © Springer Nature Switzerland AG 2024.Danicopan (Voydeya®) is an oral complement factor D inhibitor developed by Alexion AstraZeneca Rare Disease as an add-on treatment to ravulizumab or eculizumab for patients with clinically significant extravascular hemolysis. It received its first approval in Japan on 18 January 2024 for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) when used in combination with a complement component 5 (C5) inhibitor. The European Medicines Agency (EMA) also adopted a positive opinion recommending marketing authorization for danicopan for PNH patients who still experience residual hemolytic anemia despite treatment with a C5 inhibitor. This summary outlines the key milestones in danicopan's development leading to its first approval for PNH. The information presented here reflects the author's opinions. For a complete list of declarations, including funding and author disclosure statements, and copyright information, please refer to the full text online. © Springer Nature Switzerland AG 2024.