AbbVie has secured a major regulatory milestone after the US FDA approved its therapy for a rare and highly aggressive blood cancer with few effective treatment options.

 

The approval of DECNUPAZTM (pivekimab sunirine-pvzy) for adults with BPDCN is backed by results from the Phase 1/2 CADENZA trial, a global study assessing the safety and efficacy of the therapy in patients with this difficult-to-treat malignancy.

 

"For patients living with rare cancers, progress in research can be life-changing," said Roopal Thakkar, executive vice president, research and development, chief scientific officer, AbbVie. 

 

"This approval delivers a new option for treating BPDCN and demonstrates our determination to drive meaningful advancements for patients affected by difficult-to-treat cancers."

 

BPDCN typically presents with skin lesions before rapidly spreading to the bone marrow, lymph nodes, and central nervous system. It most often affects men aged 60 to 70, and despite aggressive initial treatment—including chemotherapy and sometimes stem cell transplantation—relapse is common, highlighting a persistent unmet need.

 

"BPDCN is an aggressive disease with historically limited therapeutic options, particularly for patients whose disease has relapsed or become refractory," said Naveen Pemmaraju, professor of leukemia, The University of Texas MD Anderson Cancer Center. 

 

"Pivekimab sunirine-pvzy is the first and only CD123 targeting ADC that can be initiated in an outpatient setting, offering a meaningful benefit for BPDCN patients in need of new treatment alternatives."

 

Clinical data from CADENZA showed encouraging response rates. Among newly diagnosed patients, DECNUPAZ produced a composite complete response rate of 69.7%, with a median response duration of 9.7 months. Notably, 13 patients (39.4%) were able to proceed to post-study stem cell transplantation.

 

In patients with relapsed or refractory BPDCN, the composite complete response rate was 15.7%, with a median response duration of 9.2 months. Six patients (11.8%) also went on to receive stem cell transplantation after treatment.

 

The safety profile included common adverse reactions such as edema, fatigue, musculoskeletal pain, hemorrhage, infusion-related reactions, nausea, and diarrhea. 

 

The therapy carries a boxed warning for hepatotoxicity, including hepatic veno-occlusive disease, along with additional warnings for infusion reactions, edema, sulfite allergic reactions, and embryo-fetal toxicity.