AstraZeneca shares new data on blood disorder candidate 

Dec. 11, 2023

A week after Novartis won approval for its oral paroxysmal nocturnal hemoglobinuria (PNH) treatment, AstraZeneca has unveiled long-term trial data on its own oral Factor D inhibitor, danicopan.

According to AstraZeneca, results from the 24-week and long-term extension period of the ALPHA phase 3 trial showed danicopan, when used as add-on to standard of care C5 inhibitor therapy Ultomiris or Soliris, continued to demonstrate clinical benefit for patients with PNH who experience clinically significant extravascular hemolysis (destruction of red blood cells).

The data, presented at the 65th American Society of Hematology Annual Meeting and Expo, showed that improvements in mean hemoglobin levels and absolute reticulocyte count levels, which were demonstrated at 12 weeks, were maintained through 48 weeks.

Just last week, the FDA approved Novartis' Factor B inhibitor, branded Fabhalta, as the first oral monotherapy for the treatment of adults with PNH. 

People with the rare chronic blood disorder have an acquired mutation that makes red blood cells susceptible to premature destruction by the part of the immune system known as the complement system. According to Novartis, the current standard of care, C5 inhibitor treatments, like AstraZeneca's Ultomiris or Soliris, administered as infusions, may leave symptoms, including hemolysis, uncontrolled.

Fabhalta was tested against Ultomiris or Soliris in the open-label APPLY-PNH trial, where the drug proved superiority in adults with PNH.

But, AstraZeneca's trial results suggest that a dual complement pathway inhibition at Factor D and C5 — using danicopan as an add-on to Ultomiris or Soliris — may be an optimal treatment approach for the 10-20% of patients with PNH who experience clinically significant extravascular hemolysis.

Regulatory submissions for danicopan are currently under review with multiple global health authorities.