FDA
FDA OKs Decnupaz for ultra-rare blood cancer with few treatment options
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare, fast-moving hematologic malignancy with historically limited treatment options and a high risk of relapse. Decnupaz (pivekimab sunirine-pvzy) introduces a new targeted therapy approach and becomes the first CD123-directed antibody-drug conjugate (ADC) approved for the disease that can be initiated in the outpatient setting.
On May 27, 2026, FDA approved Decnupaz (pivekimab sunirine-pvzy) for the treatment of adults with BPDCN, an ultra-rare and aggressive blood cancer that commonly affects older adults and can rapidly spread to the bone marrow, lymph nodes, and central nervous system. The drug is a CD123-directed antibody-drug conjugate designed to deliver a DNA-damaging payload directly to cancer cells expressing the CD123 protein.
Approval was supported by results from the phase 1/2 CADENZA trial, which enrolled 84 adults with either newly diagnosed or relapsed/refractory BPDCN. Among treatment-naïve patients (n=33), 69.7% achieved complete remission or clinical complete remission, with a median duration of response of 9.7 months. In the relapsed/refractory cohort (n=51), 15.7% achieved complete remission or clinical complete remission, with a median duration of response of 9.2 months. Notably, 39.4% of newly diagnosed patients and 11.8% of relapsed/refractory patients were able to proceed to stem cell transplantation after treatment.
“BPDCN is an aggressive disease with historically limited therapeutic options, particularly for patients whose disease has relapsed or become refractory,” said Naveen Pemmaraju, MD, of The University of Texas MD Anderson Cancer Center. “Pivekimab sunirine-pvzy is the first and only CD123 targeting ADC that can be initiated in an outpatient setting, offering a meaningful benefit for BPDCN patients in need of new treatment alternatives.”
The prescribing information includes a boxed warning for hepatotoxicity, including hepatic veno-occlusive disease. Common adverse reactions include edema, fatigue, musculoskeletal pain, hemorrhage, infusion-related reactions, nausea, and diarrhea.
Sources:
U.S. Food and Drug Administration. (2026, May 27). FDA approves pivekimab sunirine-pvzy for blastic plasmacytoid dendritic cell neoplasm, an ultra-rare hematologic malignancy
AbbVie Inc. (2026, May 27). U.S. FDA approves DECNUPAZ (pivekimab sunirine-pvzy) for treatment of adult patients with blastic plasmacytoid dendritic cell neoplasm, an ultra-rare and aggressive blood cancer with limited treatment options