23 October 2024 - NICE has published final evidence-based recommendations on the use of danicopan (Voydeya) in combination with ravulizumab (Ultomiris) or eculizumab (Soliris) for the treatment of adults with paroxysmal nocturnal haemoglobinuria with residual haemolytic anaemia.

Danicopan is recommended when used in combination with ravulizumab or eculizumab for the treatment of adults with paroxysmal nocturnal haemoglobinuria who have residual haemolytic anaemia, only if they have clinically significant extravascular haemolysis while on treatment with a complement component 5 inhibitor (C5 inhibitor)

Danicopan is recommended only if Alexion Pharmaceuticals provides it according to the commercial arrangement.

Read NICE technology appraisal guidance