UK – AstraZeneca’s Voydeya recommended by NICE to treat rare blood disorder PNH

AstraZeneca’s (AZ) Voydeya (danicopan) has been recommended by the National Institute for Health and Care Excellence (NICE) as an add-on therapy for adults with the rare blood disorder paroxysmal nocturnal haemoglobinuria (PNH).

The oral factor D inhibitor has been recommended for use alongside AZ’s complement component 5 (C5) inhibitors, Ultomiris (ravulizumab) or Soliris (eculizumab) to treat PNH patients who have residual haemolytic anaemia.

The health technology assessment agency also specified that those eligible for the drug will have clinically significant extravascular haemolysis (EVH) while on treatment with a C5 inhibitor and that the company must provide the drug according to the commercial arrangement…