AstraZeneca’s (AZ) Voydeya (danicopan) has been recommended by the European Medicines Agency’s human medicines committee as an add-on therapy for adults with paroxysmal nocturnal haemoglobinuria (PNH).
The oral factor D inhibitor has been specifically recommended by the Committee for Medicinal Products for Human Use (CHMP) for use alongside standard-of-care Ultomiris (ravulizumab) or Soliris (eculizumab) to treat PNH patients who have residual haemolytic anaemia.
PNH is a rare and severe blood disorder characterised by the destruction of red blood cells within blood vessels, along with white blood cells and platelet activation which can cause blood clots that can lead to organ damage.
Blocking the C5 protein helps reduce the symptoms and complications of the condition. However, up to 20% of PNH patients who are treated with a C5 inhibitor experience clinically significant extravascular haemolysis (EVH), which can result in continued symptoms of anaemia and require blood transfusions.
The CHMP’s decision on Voydeya was supported by positive results from the late-stage 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 and all key secondary endpoints, including transfusion avoidance.
Marc Dunoyer, chief executive officer of AZ’s rare disease unit, Alexion, 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 3 trial, dual complement pathway inhibition at factor D and C5 may be an optimal treatment approach for these patients.”
Voydeya was approved in Japan last month for certain adults with PNH in combination with C5 inhibitor therapy, with regulatory submissions for the therapy currently under review in additional countries.
“C5 inhibition with Ultomiris or Soliris is the standard-of-care in PNH, proven to control intravascular haemolysis and reduce life-threatening thrombotic events, yet a small portion of patients may experience clinically significant EVH,” said professor Hubert Schrezenmeier, medical director, Institute of Transfusion Medicine at the University of Ulm.
“If approved, Voydeya may optimise care for people impacted by this burdensome condition while allowing patients to maintain disease control with an established C5 inhibitor,” Schrezenmeier added.