The US Food and Drug Administration has approved pivekimab sunirine-pvzy, a CD123-directed antibody and alkylating agent conjugate, for adults with blastic plasmacytoid dendritic cell neoplasm (BPDCN), an ultrarare hematologic malignancy.
The drug had earlier received the FDA’s breakthrough designation and orphan drug designation, making the application eligible for priority review.
Efficacy was evaluated in CADENZA, a multicenter, open-label, single-arm clinical trial that included adult patients with treatment-naïve BPDCN (N=33) or relapsed or refractory BPDCN (N=51), without evidence of active central nervous system disease.
In patients with treatment-naïve BPDCN (N=33), 23 patients (69.7%) achieved complete remission or clinical complete remission (CR/CRc) with a median follow-up of 21.5 months. The median duration of CR/CRc was 9.7 months. In patients with relapsed or refractory BPDCN (N = 51), 8 patients (15.7%) achieved a CR/CRc with a median follow-up of 24.1 months. The median duration of CR/CRc was 9.2 months.
The prescribing information includes a Boxed Warning for hepatotoxicity, including hepatic veno-occlusive disease, and warnings and precautions for infusion-related reactions, edema, sulfite allergic reactions, and embryo-fetal toxicity.
The recommended pivekimab sunirine-pvzy dose is 0.045 mg/kg, calculated based on the patient’s actual body weight, administeredintravenously over approximately 15-30 minutes once every three weeks (21-day cycle) until disease progression or unacceptable toxicity.
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