Ultrarare Cancer Receives New Treatment Option
The FDA approved pivekimab sunirine-pvzy for the treatment of the rare blood cancer BPDCN.
The U.S. Food and Drug Administration (FDA) has approved pivekimab sunirine-pvzy (Decnupaz) for the treatment of adult patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare and aggressive hematologic cancer.
Pivekimab sunirine-pvzy is an antibody-drug conjugate (ADC), a targeted therapy that combines an antibody with a potent cytotoxic drug to deliver treatment to cells expressing the target antigen. The antibody portion of pivekimab sunirine-pvzy targets CD123, which is ubiquitously expressed on the surface of BPDCN cells. Once pivekimab sunirine-pvzy binds to CD123, the ADC is internalized by the cell, where it releases its cytotoxic payload, resulting in DNA damage and cell death.
Prior to this approval, treatment options for BPDCN were largely limited to intensive chemotherapy followed by stem cell transplant (SCT). However, because most patients with BPDCN are older adults and less able to tolerate toxicity associated with chemotherapy, relatively few ultimately proceed to SCT. Tagraxofusp-erzs (Elzonris) is the only other targeted therapy approved for the treatment of BPDCN. Like pivekimab sunirine-pvzy, it targets CD123; however, it differs in that CD123 is linked to a diphtheria toxin payload, which is delivered into the cancer cell and causes cell death. Although tagraxofusp-erzs established CD123 as an effective therapeutic target, additional treatment options are needed for patients whose disease progresses after treatment.
Pivekimab sunirine-pvzy was developed as an alternative CD123-directed therapy with a different payload and mechanism of action, expanding the range of available targeted treatment approaches for BPDCN. In addition to BPDCN, pivekimab sunirine-pvzy is currently being investigated for the treatment of acute myeloid leukemia, which also expresses CD123.
The approval was based on results from CADENZA, a multicenter, open-label, single-arm phase I/II clinical trial which enrolled 33 patients with BPDCN who had never received treatment and 51 patients with relapsed or refractory BPDCN, without evidence of active central nervous system disease. Efficacy was determined by the proportion of patients who experienced complete remission (CR) or clinical complete remission (CRc). CR indicates that there are no detectable signs or symptoms of disease. CRc refers to no evidence of active disease, except for residual skin changes that may persist despite successful treatment.
In previously untreated patients with BPDCN, 69.7% had a CR/CRc after a median follow-up of 21.5 months. Among patients with relapsed or refractory BPDCN, 15.7% had a CR/CRc after a median follow-up of 24.1 months. Among patients who had a CR/CRc, the response lasted at least 9.7 months in half of previously untreated patients and at least 9.2 months in half of those with relapsed or refractory disease.
The prescribing information for pivekimab sunirine-pvzy includes a Boxed Warning for hepatotoxicity, including hepatic veno-occlusive disease.
The recommended dose of pivekimab sunirine-pvzy is 0.045 mg/kg of patient body weight, administered as an intravenous infusion over approximately 15 to 30 minutes every three weeks (21-day cycle). Treatment should continue until disease progression or unacceptable toxicity occurs.
BPDCN is a rare and aggressive cancer, with an estimated incidence of approximately 0.05 cases per 100,000 individuals. Nearly 20% of patients have a prior or concurrent hematologic malignancy. According to federal statistics, the reported median survival for patients with BPDCN ranges from 8.7 to 24 months.
The FDA rendered its decision on May 27, 2026. Check this resource for updated information on all therapeutics regulated by the FDA.