NICE recommendation for new Alexion PNH treatment

danicopan shown to improve haemoglobin levels and reduce anaemia

Alexion, AstraZeneca Rare Disease, announced that the National Institute for Health and Care Excellence (NICE) has given a positive recommendation for Voydeya (danicopan).

This first-in-class, oral Factor D inhibitor can now be used as an add-on to ravulizumab or eculizumab for treating paroxysmal nocturnal haemoglobinuria (PNH) in adults with residual haemolytic anaemia, provided they have clinically significant extravascular haemolysis (EVH) while on treatment with a complement component 5 inhibitor (C5 inhibitor).

PNH is a rare, chronic, and potentially life-threatening blood disorder. It is characterised by the destruction of red blood cells within blood vessels (intravascular haemolysis), as well as the activation of white blood cells and platelets, which can result in blood clots.

Danicopan has been developed to address the needs of PNH patients who experience residual haemolytic anaemia due to clinically significant EVH while treated with a C5 inhibitor. This condition often results in continued symptoms of anaemia and may require blood transfusions.

“Today’s recommendation from NICE is a positive milestone for eligible individuals with PNH who experience the added burden of EVH symptoms such as continued anaemia, while treated with a C5 inhibitor,” said Dr Morag Griffin, Consultant Haematologist at Leeds Teaching Hospitals NHS Trust. “The ALPHA trial investigated the safety and efficacy of danicopan in patients with PNH and EVH, and demonstrates this is a suitable option to support disease control and may help to improve patients’ quality of life.”

Deborah Richards, General Manager at Alexion, AstraZeneca Rare Disease, added, “Danicopan, 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 NICE recommendation is based on the results of the pivotal ALPHA phase 3 trial.

The trial showed that danicopan, when added to ravulizumab or eculizumab, significantly improved haemoglobin levels, increased transfusion avoidance, and improved fatigue levels in patients with PNH.