North Chicago, IL – September 30, 2025 – AbbVie (NYSE: ABBV) announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for Pivekimab sunirine (PVEK), an investigational antibody-drug conjugate (ADC) developed to treat Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN), a rare and aggressive blood cancer with characteristics of both leukemia and lymphoma. The submission is based on data from the global Phase 1/2 CADENZA trial, which evaluated PVEK’s safety, tolerability, pharmacokinetics, immunogenicity, and anti-leukemia activity in patients with CD123-positive hematologic malignancies, including BPDCN and acute myeloid leukemia (AML). BPDCN often manifests with skin lesions and progresses to bone marrow, lymph nodes, and the central nervous system. Current therapies include intensive chemotherapy and stem cell transplantation, leaving a significant unmet need for innovative treatments for newly diagnosed and relapsed/refractory patients.
Science Significance
PVEK is a CD123-targeting ADC designed to deliver potent cancer cell-killing payloads directly to malignant cells expressing CD123, a protein highly overexpressed in BPDCN. This mechanism represents a precision-based approach that improves efficacy while potentially reducing systemic toxicity compared with conventional chemotherapy. The CADENZA trial identified the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) for PVEK monotherapy, providing early evidence of antileukemia activity. PVEK is also being evaluated in combination with Vidaza® (azacitidine) and Venclexta® (venetoclax) for AML, broadening its therapeutic potential. By directly targeting CD123-positive cells, PVEK exemplifies next-generation ADCs aimed at improving outcomes in rare and aggressive blood cancers.
Regulatory Significance
PVEK received Breakthrough Therapy designation from the FDA in October 2020 for relapsed/refractory BPDCN, underscoring its potential to meet a high unmet medical need. The BLA submission is a critical regulatory milestone for AbbVie and may establish PVEK as the company’s first ADC approved for blood cancers. The FDA review will evaluate whether PVEK provides meaningful clinical benefit relative to existing therapies, which could pave the way for timely access to a novel treatment for patients with limited options. This milestone highlights regulatory pathways that support the accelerated development of innovative therapies for rare diseases.
Business Significance
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The BLA submission reinforces AbbVie’s commitment to expanding its oncology portfolio, particularly in hematologic malignancies. With over 35 investigational oncology medicines in development, the company is advancing therapies across small molecules, ADCs, immuno-oncology agents, multispecific antibodies, and CAR-T platforms. PVEK strengthens AbbVie’s presence in rare blood cancers and underscores the company’s strategic focus on targeted, precision oncology therapies. Successful approval could provide significant commercial opportunities, enhance AbbVie’s competitive positioning in ADC development, and reinforce its leadership in delivering transformative oncology therapies globally.
Patients’ Significance
BPDCN patients face limited treatment options, poor prognosis, and significant unmet medical needs, especially for those relapsed after chemotherapy or ineligible for stem cell transplant. PVEK offers a novel, precision-targeted approach, delivering cytotoxic payloads directly to CD123-positive cells, potentially improving clinical outcomes and quality of life. The therapy may provide safer and more effective alternatives to conventional treatment, representing a meaningful advance for this underserved patient population. For patients living with rare blood cancers, PVEK may be a transformative option that could alter disease progression and treatment experience.
Policy Significance
The BLA submission aligns with policy initiatives promoting accelerated development for rare and high unmet need diseases. PVEK exemplifies innovation in antibody-drug conjugates and precision oncology, supporting frameworks that expedite review while maintaining safety and efficacy. Ensuring timely patient access to breakthrough therapies is a public health priority, and PVEK may set precedents for future regulatory guidance and policy in rare disease treatment development. This submission reflects ongoing efforts to facilitate innovation and improve healthcare outcomes for underserved populations globally.
Transaction Highlights
AbbVie’s submission of the Biologics License Application (BLA) for Pivekimab sunirine (PVEK) underscores the company’s strategic commitment to advancing antibody-drug conjugates (ADCs) and precision oncology therapeutics. PVEK is being evaluated both as monotherapy in BPDCN and in combination with Vidaza® (azacitidine) and Venclexta® (venetoclax) for patients with acute myeloid leukemia (AML), reflecting AbbVie’s focus on developing multi-indication ADCs that can address several hematologic malignancies. The CADENZA trial provided positive safety and efficacy data, establishing the maximum tolerated dose and recommended Phase 2 dose, which form a strong foundation for regulatory approval. This milestone highlights AbbVie’s capability to leverage innovative ADC technology to expand its oncology pipeline and reinforces its competitive positioning in the evolving blood cancer treatment market. By advancing PVEK toward potential FDA approval, AbbVie is strategically positioning itself to capture growth in rare and high unmet need hematologic cancers while strengthening its leadership in targeted cancer therapies globally.
Source: AbbVie Press Release
