AbbVie continues to make strides in oncology with the recent FDA approval of a medication targeting an ultra-rare and aggressive form of blood cancer that has limited treatment options. This latest development concerns pivekimab sunirine, which is indicated for adults diagnosed with blastic plasmacytoid dendritic cell neoplasm (BPDCN).
The FDA’s decision, announced on Wednesday, clears the way for AbbVie to market pivekimab sunirine, which will be administered intravenously under the brand name Decnupaz. BPDCN originates in the bone marrow and blood, spreading to various organs, the central nervous system, and the skin. Notably, patients suffering from BPDCN often present with dark purple skin lesions and are typically aged 60 and older, with a higher incidence in men than women.
Currently, chemotherapy is the standard treatment for BPDCN. Research has revealed that BPDCN cells overexpress a protein known as CD123, paving the way for targeted therapies. In 2018, Stemline Therapeutics, now a subsidiary of Menarini Group, received FDA approval for Elzonris, another treatment aimed at CD123. Following targeted therapy, patients may undergo stem cell transplants; however, relapses remain a common challenge.
The approval of Decnupaz provides BPDCN patients with a new targeted therapeutic option. This antibody-drug conjugate (ADC) is specifically designed to target CD123. Clinical evaluation included an open-label phase 1/2 study involving adults whose BPDCN was positive for the target protein. Among a cohort of 33 treatment-naive patients, 69.7% (23 patients) achieved complete or clinical remission, with a median follow-up of 21.5 months. Similarly, in a cohort of 51 patients with relapsed or refractory BPDCN, 15.7% (eight patients) achieved complete or clinical remission, with a median follow-up of 24.1 months.
“This approval offers a new treatment option for BPDCN and reflects our commitment to making significant advancements for patients faced with difficult-to-treat cancers,” stated Roopal Thakkar, executive vice president, research and development, and chief scientific officer at AbbVie, in a prepared statement.
Common adverse reactions reported in the BPDCN clinical trial included edema, fatigue, myalgia, hemorrhage, and infusion-related reactions. The labeling for Decnupaz includes a boxed warning regarding veno-occlusive disease, a severe liver toxicity that can be fatal. Clinicians are advised to monitor for signs of this complication and conduct liver health assessments before each intravenous infusion.
Decnupaz was part of AbbVie’s $10 billion acquisition of ADC specialist ImmunoGen. This 2023 deal featured Elahere, which gained distinction as the first ADC approved for ovarian cancer under ImmunoGen. At the time of the acquisition, Decnupaz had reached the Phase 2 segment of its ongoing trial.
AbbVie’s own ADC research has made significant progress as well. Last year, Emrelis received accelerated FDA approval for non-small cell lung cancer, targeting the c-Met protein. The company holds optimistic expectations for teisotuzumab adizutecan, or Temab-A, described as a next-generation ADC targeting c-Met, with clinical trials covering multiple solid tumors, including gastroesophageal adenocarcinoma and non-small cell lung cancer. AbbVie is set to present early clinical data for Temab-A in ovarian cancer and head and neck cancer at the upcoming American Society of Clinical Oncology annual meeting in Chicago.
In the meantime, AbbVie is exploring potential additional applications for Decnupaz in treating other types of blood cancers, with a phase 2 trial ongoing in acute myeloid leukemia.
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