NICE recommends Voydeya for PNH

UK pharma major AstraZeneca’s (LSE: AZN) Alexion Rare Disease unit has received a positive recommendation for Voydeya (danicopan), from the UK’s National Institute for Health and Care Excellence (NICE).

The health technology assessor has backed Voydeya as an add-on to AstraZeneca’ ravulizumab (Ultomiris) or eculizumab (Soliris) as an option for treating paroxysmal nocturnal hemoglobinuria (PNH) in adults who have residual hemolytic anemia only if they have clinically significant extravascular hemolysis (EVH) while on treatment with a complement component 5 inhibitor (C5 inhibitor) and the company provides it according to the commercial arrangement.1

Danicopan is a first-in-class, oral, Factor D inhibitor, developed as an add-on to standard-of-care ravulizumab or eculizumab to address the needs of a small number of eligible patients with PNH who experience residual hemolytic anemia due to clinically significant EVH while treated with a C5 inhibitor. Clinically significant EVH results in continued symptoms of anemia and may require blood transfusions.