AbbVie Submits FDA BLA For Pivekimab Sunirine In Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) Treatment, A Rare Blood Cancer Therapy

AbbVie,  has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking approval for Pivekimab sunirine (PVEK), an investigational therapy for Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN). The application is supported by results from the Phase 1/2 CADENZA trial, a global study assessing the safety and efficacy of PVEK in patients with this rare and aggressive blood cancer.

"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. 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 (BPDCN)," said Roopal Thakkar, executive vice president, research and development and chief scientific officer, AbbVie. 

BPDCN is characterized by features of both leukemia and lymphoma and often presents with skin lesions, while spreading to the bone marrow, central nervous system, and lymph nodes. Current treatment approaches rely on intensive chemotherapy, often followed by stem cell transplantation, but relapse rates remain high—creating a significant unmet medical need for novel therapeutic options.

PVEK is a CD123-targeting Antibody-Drug Conjugate (ADC) designed to selectively deliver potent anti-cancer agents to cells expressing the CD123 protein, which is overexpressed in BPDCN. Beyond BPDCN, PVEK is also in development for acute myeloid leukemia (AML), being studied as a monotherapy in BPDCN and in combination with Vidaza® (azacitidine) and Venclexta® (venetoclax) in AML. The therapy previously received FDA Breakthrough Therapy designation in October 2020 for relapsed or refractory BPDCN.