WASHINGTON, May 29, 2026 – The U.S. Food and Drug Administration (FDA) recently approved pivekimab sunirine‑pvzy (Decnupaz™) for adults with blastic plasmacytoid dendritic cell neoplasm (BPDCN), an ultra‑rare and aggressive blood cancer.
“This is meaningful for patients with BPDCN, where choices have historically been limited, especially for those whose disease has relapsed or stopped responding to prior treatments” said Lore Gruenbaum, Ph.D., Chief Scientific Officer of Blood Cancer United.
The approval was based on results from the CADENZA clinical trial, which enrolled adults with BPDCN and no evidence of active central nervous system disease.
Among 33 patients in the trial who had not previously received treatment, nearly 70% responded to pivekimab sunirine‑pvzy treatment and 13 patients went on to receive a potentially curative stem cell transplant.
Among 51 patients in the trial who had received prior treatment, just under 16% responded to pivekimab sunirine‑pvzy and 6 patients went on to stem cell transplant.
Decades of Investment in Foundational Science
The approval of pivekimab sunirine‑pvzy builds on decades of foundational BPDCN research that Blood Cancer United helped advance through sustained investment in scientists and discovery at a time when the disease was poorly understood and frequently unrecognized.
Beginning in the late 1990s, Blood Cancer United supported foundational studies by Arthur Frankel, M.D., whose early studies of CD123‑targeted approaches helped establish BPDCN as a distinct disease entity and laid critical groundwork for the development of tagraxofusp-erzs (Elzonris), the first FDA‑approved therapy for BPDCN.
Starting in 2013, Blood Cancer United provided critical financial support for the pivotal clinical trial for tagraxofusp through its Therapy Acceleration Program (TAP), a venture philanthropy program.
Blood Cancer United also played a central role in supporting the work of Andy Lane, M.D., Ph.D., whose research has expanded the understanding of BPDCN biology and management.
Together, these long‑term investments helped move BPDCN from a neglected diagnosis to a disease with growing treatment options.
“New approvals like this underscore how sustained support for research from the laboratory all the way to clinical trials is needed to bring meaningful advances to patients with even the rarest blood cancers,” Dr. Gruenbaum concluded.
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