The FDA has approved Pivekimab sunirine-pvzy (Decnupaz) for adults with blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare and aggressive blood cancer. The drug is the first CD123-targeting antibody-drug conjugate that can be started in an outpatient setting. It is administered intravenously every three weeks based on actual body weight.
Approval was supported by results from the phase 1/2 CADENZA trial. Among treatment-naive patients, the therapy achieved a complete remission or clinical complete remission rate of 69.7%, with a median response duration of 9.7 months. Nearly 40% of these patients were able to proceed to stem cell transplantation.
In relapsed or refractory BPDCN, 15.7% of patients achieved remission, with a median response duration of 9.2 months and median overall survival of 5.8 months.
The treatment carries a boxed warning for severe hepatotoxicity, including hepatic veno-occlusive disease. Common side effects included edema, fatigue, musculoskeletal pain, hemorrhage, infusion reactions, nausea, and diarrhea.