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FDA Approves Decnupaz for Rare Blood Cancer

The approval gives adults with blastic plasmacytoid dendritic cell neoplasm a second CD123‑targeted treatment backed by CADENZA response data, with a boxed warning for serious liver toxicity.

Overview

  • The U.S. Food and Drug Administration cleared pivekimab sunirine (Decnupaz) for use in adults with blastic plasmacytoid dendritic cell neoplasm, a rare and aggressive hematologic cancer.
  • Regulatory backing rested on the single‑arm CADENZA trial of 84 adults in which 69% of previously untreated patients achieved a composite complete response and 15.7% of relapsed or refractory patients achieved CR/CRc.
  • Median duration of complete remission was roughly 9 to 10 months in both the frontline and relapsed cohorts, with median follow‑up times of about 21.5 and 24.1 months respectively.
  • The drug label carries a boxed warning for hepatotoxicity, including hepatic veno‑occlusive disease, and additional precautions for infusion reactions, edema, sulfite allergic reactions, and embryo‑fetal toxicity.
  • Pivekimab targets CD123, a protein commonly overexpressed on BPDCN cells, and joins tagraxofusp as the only FDA‑approved therapies for this disease, offering a new frontline option but requiring careful liver monitoring in treated patients.