On May 27, the U.S. Food and Drug Administration (FDA) approved pivekimab sunirine-pvzy (Decnupaz), a CD123-directed antibody and alkylating agent conjugate, for adults with blastic plasmacytoid dendritic cell neoplasm (BPDCN).
CADENZA
Efficacy was evaluated in CADENZA (ClinicalTrials.gov identifier NCT03386513), a multicenter, open-label, single-arm clinical trial that included treatment-naive adult patients with BPDCN (n = 33) or relapsed or refractory BPDCN (n = 51), without evidence of active central nervous system disease. Efficacy was based on the rate of complete remission or clinical complete remission.
Among the 33 patients with treatment-naive BPDCN, 23 (69.7%; 95% confidence interval [CI] = 51.3%–84.4%) achieved a complete remission or clinical complete remission with a median follow-up of 21.5 months. The median duration of complete remission or clinical complete remission was 9.7 months (95% CI = 2.9 months to not estimable). Among the 51 patients with relapsed or refractory BPDCN, 8 (15.7%; 95% CI = 7.0%–28.6%) achieved a complete remission or clinical complete remission with a median follow-up of 24.1 months. The median duration of complete remission or clinical complete remission was 9.2 months (range = 2.7 to 27.6+ 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.
Recommended Dosage
The recommended pivekimab sunirine-pvzy dose is 0.045 mg/kg intravenously over approximately 15 to 30 minutes once every 3 weeks (21-day cycle) until disease progression or unacceptable toxicity. The dose should be calculated based on the patient’s actual body weight.
This review used the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment.
This application was granted Priority Review. Pivekimab sunirine-pvzy received Breakthrough Therapy designation and Orphan Drug designation.
