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(Alliance News) - AstraZeneca PLC on Monday reported "positive results" from a test of danicopan for the treatment of paroxysmal nocturnal haemoglobinuria, or PNH, a rare and severe blood disorder.
The pharmaceutical maker said danicopan was tested as an add-on to to standard of care C5 inhibitor therapy Ultomiris or Soliris, whose generic names are ravulizumab and eculizumab.
PNH is characterised by the destruction of red blood cells within blood vessels, known as intravascular haemolysis, or IVH, and white blood cell and platelet activation that can cause blood clots and result in organ damage and death.
The 24-week and long-term extension period of the Alpha phase III trial showed that danicopan as an add-on showed clinical benefit for patients with PNH who experience clinically significant extravascular haemolysis, or EVH, which can result in continued symptoms of anaemia and require blood transfusions.
Improvements in mean haemoglobin levels and absolute reticulocyte count levels, which were demonstrated at 12 weeks, were maintained through 48 weeks, Astra said.
"Unlike IVH, EVH is not life-threatening, but its manifestations can be burdensome for people living with this condition, which is why we continue to explore the potential of the complement system to advance patient care," explained Gianluca Pirozzi, head of development, Regulatory & Safety, at Astra division Alexion.
AstraZeneca shares were up 0.1% at 10,128.00 pence early Monday in London.
By Tom Waite, Alliance News editor
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