Pivekimab Sunirine Application Submitted to FDA for Rare Blood Cancer

A biologics license application for pivekimab sunirine (PVEK) has been submitted to the U.S. Food and Drug Administration (FDA) for the treatment for blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare blood cancer, according to a news release from AbbVie.

The submission for PVEK is based on results from the global phase 1/2 CADENZA trial, which showed high and lasting remission rates in adults with BPDCN, according to study findings published in Journal of Clinical Oncology. Treatment was generally tolerable, side effects were manageable, and no capillary leak syndrome occurred.

The median follow-up was 21.5 months for patients receiving frontline treatment and 24.1 months for those with relapsed or refractory disease.

Among patients treated first-line, the combined rate of complete remission and complete remission with minimal skin abnormality was 70%, and the median duration of response for complete remission and complete remission with minimal skin abnormality was 9.8 months. The overall response rate was 85%, and the median overall survival was 16.6 months. In frontline patients who went on to receive stem cell transplants, complete remission and complete remission with minimal skin abnormality was 92%, and median overall survival was not reached.

For patients with relapsed or refractory BPDCN, complete remission and complete remission with minimal skin abnormality was 14%, with a median duration of response of 9.2 months. The overall response rate was 35%, median overall survival was 5.8 months, and 12% of patients were able to receive a stem cell transplant.

Patients in the frontline group received a median of 5 cycles of PVEK, while the relapsed or refractory group received a median of 3 cycles.

"Meaningful innovations in cancer research and treatment are happening every day. It is important that these innovations reach patients who desperately need them, including those with rare cancers who have limited options,” Roopal Thakkar, executive vice president, research and development and chief scientific officer, AbbVie, said in the news release.

He continued, "We look forward to next steps in the regulatory process for our latest antibody-drug conjugate (ADC), our first ADC in blood cancer, and how it may advance treatment for those living with blastic plasmacytoid dendritic cell neoplasm."

Safety was evaluated in all 84 patients in the CADENZA trial. The most common side effect was peripheral edema, occurring in 54% of patients and in 12% at more severe levels (grade 3 [severe] or higher). Side effects led to stopping treatment in 9% of frontline patients and 7% of those with relapsed or refractory disease.

No cases of capillary leak syndrome or treatment-related deaths were reported. Two patients (2%) experienced veno-occlusive disease of the liver after cycles 4 and 8, which resolved.

Trial Design of the Phase 1/2 CADENZA Trial

Adults with BPDCN received PVEK as an intravenous (into the vein) infusion lasting less than 30 minutes on day 1 of a 21-day cycle. The primary goal was the rate of composite complete response, combining CR and CRc in frontline patients. Key secondary measures included duration of CR and CRc, overall survival, overall response rate, the percentage of patients bridged to stem cell transplant after PVEK and safety and tolerability.

What is Blastic Plasmacytoid Dendritic Cell Neoplasm? How Does PVEK Work?

BPDCN is a rare, aggressive blood cancer with features of both leukemia and lymphoma. Patients often develop skin lesions, and the disease can spread to the bone marrow, central nervous system, and lymph nodes, as per the release. First-line treatment usually involves intensive chemotherapy, often followed by stem cell transplant, but additional and innovative options are needed for newly diagnosed patients and those with relapsed or refractory disease.

PVEK is an antibody-drug conjugate that targets CD123, a protein overexpressed in BPDCN. This therapy delivers a potent cancer-killing payload directly to cells with CD123, offering a promising approach for patients with BPDCN and other blood cancers such as acute myeloid leukemia.

References

1. “AbbVie Submits Biologics License Application (BLA) to U.S. FDA for Pivekimab sunirine (PVEK) - an Investigational Antibody-Drug Conjugate (ADC) to Treat Rare Cancer with Limited Treatment Options.” News Release. Abbvie. Sep 30. 2025. https://ascopubs.org/doi/10.1200/JCO.2025.43.16_suppl.6502
2. “Efficacy and safety of pivekimab sunirine (PVEK) in patients (pts) with blastic plasmacytoid dendritic cell neoplasm (BPDCN) in the CADENZA study,” by Dr. Naveen Pemmaraju, et al., Journal of Clinical Oncology. https://news.abbvie.com/2025-09-30-AbbVie-Submits-Biologics-License-Application-BLA-to-U-S-FDA-for-Pivekimab-sunirine-PVEK-an-Investigational-Antibody-Drug-Conjugate-ADC-to-Treat-Rare-Cancer-with-Limited-Treatment-Options

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