Decnupaz Approved for Rare Blood Cancer

The FDA approved the antibody-drug conjugate pivekimab sunirine (Decnupaz) for the rare hematologic malignancy blastic plasmacytoid dendritic cell neoplasm (BPDCN).

The approval stipulates use of the drug in adults. Although BPDCN occurs most often in patients ages 60 and older, the condition can affect patients in any age group.

Primary support for the approval came from the prospective, single-arm, open-label CADENZA trial involving 84 adults. The study population comprised 33 patients with previously untreated BPDCN (22 with de novo disease and 11 with co-existing hematologic malignancy) and 51 with relapsed/refractory disease. The primary endpoint was composite complete response (CCR), defined as complete remission (CR) plus investigator-assessed clinical complete remission (CRc) in patients who received pivekimab as frontline therapy.

After a median follow-up of 21.5 months among those with previously untreated BPDCN, 69% met the primary endpoint of CCR. Median duration of CR/CRc was 9.7 months. In the group with relapsed/refractory disease, 15.7% achieved CR/CRc after a median follow-up of 24.1 months, and median duration of CR/CRc was 9.2 months.

“These strong, durable response results offer hope to BPDCN patients with limited treatment options,” principal investigator Naveen Pemmaraju, MD, of the University of Texas MD Anderson Cancer Center in Houston, said in a statement when the results were published. “An effective and safe frontline treatment for patients would be practice changing, and these positive results suggest that [pivekimab] should be considered a potential standard treatment for BPDCN patients.”

BPDCN affects an estimated 500-1,000 patients annually in the U.S. The condition is highly aggressive, involving multiple organ sites that include skin, bone marrow, lymph nodes, and extra-nodal sites in some cases, and has a poor prognosis. The CD123 antigen is overexpressed in BPDCN cells, providing the rationale for development of pivekimab sunirine, an anti-CD123 antibody linked to a cytotoxic agent.

Pivekimab joins tagraxofusp (Elzonris), a CD123-targeted cytotoxin, as the only FDA-approved therapies for BPDCN.

Prescribing information for pivekimab includes a boxed warning for hepatotoxicity, including hepatic veno-occlusive disease, as well as warnings and precautions for infusion-related reactions, edema, sulfite allergic reactions, and embryo-fetal toxicity.