More Phase III success for rare blood disorder franchise

16 September 2022

Looking to cement its lead in the treatment of rare blood disorders, AstraZeneca (LSE: AZN) has lifted the lid on positive results for a new add-on therapy for its blockbuster C5 inhibitor franchise.

Danicopan is being developed initially for people with paroxysmal nocturnal hemoglobinuria (PNH), to go alongside Ultomiris (ravulizumab) or Soliris (eculizumab).

The oral factor D inhibitor could be used to help people with PNH who experience extravascular hemolysis, the abnormal breakdown of red blood cells.

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