ASH 2024: New Data in Paroxysmal Nocturnal Hemoglobinuria

Ilene Weitz, MD

Disclosures

November 26, 2024

Ilene Weitz, MD, details a few abstracts on paroxysmal nocturnal hemoglobinuria (PNH) treatments that will be presented at ASH 2024, particularly data on danicopan as an add-on to ravulizumab or eculizumab from the phase 3 ALPHA trial. This study demonstrates that danicopan significantly improves hemoglobin stability, fatigue, and overall quality of life for patients with PNH with extravascular hemolysis.

In addition, Dr Weitz highlights the APPLY-PNH trial on iptacopan monotherapy, which showed improved physical functioning and reduced symptoms at both 24 and 48 weeks.

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