Danicopan (Voydeya®) is an oral complement factor D inhibitor being developed by Alexion AstraZeneca Rare Disease as an add-on treatment to ravulizumab or eculizumab for patients with clinically significant extravascular haemolysis. It recently received its first approval in Japan on January 18, 2024, for the treatment of adults with paroxysmal nocturnal haemoglobinuria when used in combination with a complement C5 inhibitor. Subsequently, the European Medicines Agency adopted a positive opinion recommending the granting of marketing authorization for danicopan for patients with paroxysmal nocturnal haemoglobinuria who continue to have residual haemolytic anaemia despite treatment with a complement component 5 inhibitor. This article summarizes the key milestones in the development of danicopan leading to this first approval.Danicopan (Voydeya®) is an oral complement factor D inhibitor being developed by Alexion AstraZeneca Rare Disease as an add-on treatment to ravulizumab or eculizumab for patients with clinically significant extravascular haemolysis. It recently received its first approval in Japan on January 18, 2024, for the treatment of adults with paroxysmal nocturnal haemoglobinuria when used in combination with a complement C5 inhibitor. Subsequently, the European Medicines Agency adopted a positive opinion recommending the granting of marketing authorization for danicopan for patients with paroxysmal nocturnal haemoglobinuria who continue to have residual haemolytic anaemia despite treatment with a complement component 5 inhibitor. This article summarizes the key milestones in the development of danicopan leading to this first approval.