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AZ’s Danicopan Could Protect Ultomiris After Pivotal Combination Success In PNH

 
• By Ayisha Sharma

The major’s factor D inhibitor has increased hemoglobin in a subset of paroxysmal nocturnal hemoglobinuria patients when added to a C5 inhibitor, paving the way for an approval that could help protect Ultomiris from competition.  

woman in yellow top looking tired
PNH Patients With EVH Experience Fatigue, Among Other Symptoms • Source: Shutterstock
Key Takeaways:

  • Up to 20% of PNH patients have clinically significant extravascular hemolysis despite C5 inhibitor treatment.

  • When added to a C5 inhibitor, danicopan increased hemoglobin by an average of 2.94g/dL.

  • Danicopan is also being studied as a monotherapy for geographic atrophy.

AstraZeneca PLC’s danicopan has succeeded in a Phase III trial of paroxysmal nocturnal hemoglobinuria (PNH) patients experiencing extravascular hemolysis (EVH) despite standard of

PNH is a rare and severe blood disorder estimated to affect between one and nine per million people

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