AstraZeneca's danicopan shows benefit in rare blood disorder, meets trial's main goal
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AstraZeneca (NASDAQ:AZN) said its investigational drug danicopan as an add-on to Ultomiris or Soliris showed positive high-level results in patients with paroxysmal nocturnal haemoglobinuria (PNH) who experience clinically significant extravascular haemolysis (EVH) in a phase 3 trial.
PNH is a rare disorder in which red blood cells break apart prematurely. EVH is the destruction of red blood cells outside the circulatory system, mostly in the liver and spleen.
An interim analysis of the phase 3 trial dubbed, ALPHA, showed that danicopan (ALXN2040) added to Ultomiris (ravulizumab) or Soliris (eculizumab) met the main goal of change in hemoglobin from baseline at 12 weeks, showing statistically significant and clinically meaningful improvements in hemoglobin levels, compared to placebo plus Ultomiris or Soliris, the company said in a Sept. 16 press release.
The British pharma giant added that the drug also met key secondary goals, by showing improvements in transfusion avoidance and change in Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue score.
"These are the first positive Phase III results for an oral factor D inhibitor and demonstrate the potential for danicopan add-on therapy to improve signs and symptoms and reduce the need for transfusions for the limited proportion of people living with PNH who experience clinically significant EVH," said Alexion CEO Marc Dunoyer.
The comany added that danicopan was generally well tolerated and there were no clinically meaningful differences in safety results seen between the danicopan plus C5 inhibitor group and control group.
AstraZeneca said that it plans to proceed with regulatory submissions in the coming months.