AstraZeneca’s (AZ) Voydeya (danicopan) has been approved by the European Commission (EC) as an add-on therapy for adults with paroxysmal nocturnal haemoglobinuria (PNH).
The oral factor D inhibitor has been authorised for use alongside AZ’s complement C5 inhibitors, Ultomiris (ravulizumab) or Soliris (eculizumab), to treat PNH patients with residual haemolytic anaemia.
PNH is a rare and severe blood disorder that can result in a range of signs and 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, up to 20% of PNH patients who are treated with a C5 inhibitor experience clinically significant extravascular haemolysis (EVH), which results in continued symptoms of anaemia and may require blood transfusions.
The EC’s decision on Voydeya follows a recent recommendation from the European Medicines Agency’s human medicines committee and 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 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.
Marc Dunoyer, chief executive officer of Alexion, AZ’s rare disease unit, said: “For patients with PNH, Voydeya as an add-on therapy has been shown to address signs and symptoms of clinically significant EVH, including anaemia, while maintaining standard-of-care treatment with Soliris or Ultomiris.
“We look forward to making this first-in-class Factor D inhibitor available to patients in Europe and to advancing access around the globe.”
The approval comes just over three weeks after the US Food and Drug Administration approved Voydeya as an add-on therapy for adult PNH patients experiencing EVH.
The therapy has also been authorised for use in Japan for certain adults with PNH and regulatory submissions are currently under review in additional countries.
Beyond PNH, Voydeya is being evaluated in a mid-stage clinical trial as a potential monotherapy for geographic atrophy, an advanced form of age-related macular degeneration.