AstraZeneca’s Voydeya granted FDA approval for rare blood disease PNH
AstraZeneca’s (AZ) Voydeya (danicopan) has been approved by the US Food and Drug Administration (FDA) as an add-on therapy for paroxysmal nocturnal haemoglobinuria (PNH). The oral factor D has been specifically authorised for use alongside AZ’s complement C5 inhibitors Ultomiris (ravulizumab) or Soliris (eculizumab) to treat extravascular haemolysis (EVH) in adults with PNH.
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.
Immediate, complete and sustained terminal complement inhibition by blocking the C5 protein helps reduce symptoms and complications of the condition. However, up to 20% of PNH patients who are treated with a C5 inhibitor experience clinically significant EVH, which can result in continued symptoms of anaemia and require blood transfusions.
The FDA’s decision on Voydeya was supported by positive results from the late-stage ALPHA trial, which evaluated the efficacy and safety of the therapy 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.
The approval comes just one week after Voydeya was recommended by the European Medicines Agency’s human medicines committee as an add-on therapy for PNH patients with residual haemolytic anaemia.
The therapy has also been approved in Japan for certain adults with PNH and regulatory submissions are currently under review in additional countries.
Commenting on the latest authorisation, Bart Scott, of the University of Washington Medical Center and Fred Hutchinson Cancer Center, said: “The approval of Voydeya offers this small subset of PNH patients an add-on therapy designed to address EVH while maintaining disease control with Ultomiris or Soliris.”
April 3, 2024
