EUROPE—The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended approval of AstraZeneca’s oral Factor D inhibitor Voydeya (danicopan) for treatment of paroxysmal nocturnal hemoglobinuria (PNH).
The decision was based on The ALPHA Phase III clinical trial, a worldwide, double-blind, multiple-dose, placebo-controlled investigation aimed at assessing the safety and effectiveness of Voydeya as a supplement to C5 inhibitor therapy in PNH patients with clinically significant extravascular hemolysis (EVH).
In addition to their continued Ultomiris or Soliris medication, trial participants were randomly assigned to receive Voydeya or a placebo in a 2:1 ratio for a duration of 12 weeks. Voydeya achieved all significant secondary objectives as well as the primary endpoint, which was the change in hemoglobin levels from baseline to week 12.
The secondary goals were avoiding transfusions and differences in patients’ Functional Assessment of Chronic Illness Therapy – Fatigue score.
Overall, voydeya was well accepted, and no new safety issues were noted in regard to treating the rare, acquired, life-threatening disease of the blood which is characterized by destruction of red blood cells, blood clots, and impaired bone marrow function.
“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,” stated Marc Dunoyer, CEO of AstraZeneca Rare Disease group Alexion.
“Dual complement pathway inhibition at Factor D and C5 may be an ideal treatment approach for these patients, as we observed in the pivotal ALPHA Phase III trial.” He added.
Earlier, AstraZeneca concluded the USS$1.2 billion acquisition of Gracell Biotechnologies, a clinical-stage biopharmaceutical business located in China.
Following the acquisition, Gracell, which has a presence in China and US markets, will become an entirely owned subsidiary of AstraZeneca.
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