The FDA has approved pivekimab sunirine-pvzy (Decnupaz) for adults with blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare blood cancer. The first-in-class antibody-drug conjugate targets CD123 and delivers a DNA-damaging payload directly to cancer cells.
Approval was based on the Phase 1/2 CADENZA trial. In treatment-naive patients, the complete remission rate reached 69.7%, with a median overall survival of 16.6 months. Previously treated patients had a lower remission rate of 15.7% and median overall survival of 5.8 months. Twelve-month overall survival for untreated patients was 64%.
The approved dose is 0.045 mg/kg intravenously every three weeks.
The drug carries a boxed warning for hepatotoxicity, including hepatic veno-occlusive disease. Common side effects included peripheral edema, fatigue, infusion reactions, nausea, and hypokalemia. Severe treatment-related adverse events occurred in 36% of patients, while 13% discontinued treatment due to toxicity.