The US Food and Drug Administration (FDA) has approved pivekimab sunirine-pvzy for adults with blastic plasmacytoid dendritic cell neoplasm (BPDCN).
BPDCN is a rare, aggressive CD4+ CD56+ myeloid cancer characterized by cutaneous lesions, bone marrow involvement, and leukemic dissemination. The condition was previously known as acute agranular CD4+ natural killer (NK) cell leukemia. Unfortunately, despite treatment, relapse is common, and overall survival rates are poor. In adults, allogeneic stem cell transplantation improves overall survival during initial remission.
The approval of pivekimab sunirine is based on safety and efficacy data from the multicenter, open-label, single-arm CADENZA (NCT03386513) clinical trial that included 33 adult patients with treatment-naïve BPDCN and 51 adult patients with relapsed or refractory BPDCN, without evidence of active central nervous system disease. Pivekimab sunirine is a CD123-directed antibody and alkylating agent conjugate. Efficacy was based on the rate of complete remission or clinical complete remission (CR/CRc).
In patients with treatment-naïve BPDCN, 23 patients achieved a 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, 8 patients 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.
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