The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has recommended AstraZeneca's oral Factor D inhibitor, Voydeya (danicopan), for marketing authorisation to treat paroxysmal nocturnal haemoglobinuria (PNH).

The product is an additional treatment to standard-of-care Ultomiris (ravulizumab) or Soliris (eculizumab) for adult PNH patients who have residual haemolytic anaemia.

The positive opinion is based on data from the ALPHA Phase III clinical trial, a global, double-blind, multiple-dose, placebo-controlled study designed to evaluate the safety and efficacy of Voydeya as an add-on to a C5 inhibitor therapy in PNH patients with clinically significant extravascular haemolysis (EVH).

Patients in the trial were randomised to receive either Voydeya or a placebo in a 2:1 ratio, in addition to their ongoing Soliris or Ultomiris therapy, for 12 weeks.

Voydeya met the primary endpoint of variation in haemoglobin levels from baseline to week 12, and all key secondary endpoints.

Transfusion avoidance and variation in patients' Functional Assessment of Chronic Illness Therapy – Fatigue score were among the secondary endpoints.

Voydeya was generally well tolerated with no new safety concerns reported.

AstraZeneca Rare Disease group Alexion CEO Marc Dunoyer said: “The positive CHMP recommendation recognises the promise of Voydeya as an add-on to standard-of-care to address signs and symptoms of clinically significant EVH for this small subset of patients.

“As we saw in the pivotal ALPHA Phase III trial, dual complement pathway inhibition at Factor D and C5 may be an optimal treatment approach for these patients.”

The latest development comes after AstraZeneca concluded the acquisition of China-based clinical-stage biopharmaceutical company Gracell Biotechnologies for $1.2bn.

The acquisition, initially agreed in December 2023, positions Gracell as a wholly owned subsidiary of AstraZeneca with operations continuing in the US and China.