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.
PNH is caused by an acquired genetic mutation and results in the body’s immune system attacking its red blood cells. Patients can experience a range of symptoms, including blood clots, abdominal pain, difficulty swallowing, shortness of breath, excessive fatigue and anaemia.
The symptoms and complications of the condition can be reduced by blocking the C5 protein. However, a subset of PNH patients who are treated with a C5 inhibitor experience clinically significant EVH, which results in continued symptoms of anaemia and may require blood transfusions.
NICE’s decision on Voydeya was supported by positive results from the phase 3 ALPHA trial, which evaluated the efficacy and safety of the drug as an add-on to Ultomiris or Soliris in patients with PNH who experienced clinically significant EVH.
Results showed that Voydeya met the primary endpoint of change in haemoglobin from baseline to week 12 as well as all key secondary endpoints, including transfusion avoidance and change in FACIT-Fatigue score, which measures patients’ level of fatigue during their usual daily activities.
Deborah Richards, general manager of Alexion, AZ’s rare disease unit, said: “[Voydeya], a first-in-class, oral, factor D inhibitor, is the latest innovation in decades of work in our complement inhibition research programme. We’re committed to bettering the lives of all patients with PNH and we look forward to making this treatment available to eligible patients across the country.”
The positive opinion comes six months after the European Commission approved Voydeya as an add-on therapy for adult PNH patients with residual haemolytic anaemia. The therapy has also been authorised for use in the US and Japan for certain adults with PNH.
