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Naveen Pemmaraju, MD: Tagraxofusp for BPDCN

New trial results show the cytotoxin delivers clinical responses in patients with untreated or relapsed forms of the rare disease.

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare, frequently misdiagnosed, and aggressive hematologic malignancy that affects fewer than 1000 patients annually. It wasn’t until recently that clinicians had resources—and, to an extent, genuine hope—to combat the rare disease.

In an interview with MD Magazine® while at the American Society of Clinical Oncology (ASCO) 2019 Annual Meeting in Chicago, IL, Naveen Pemmaraju, MD, of the Department of Medicine, Division of Leukemia at MD Anderson Center, detailed the promising findings of recently US Food and Drug Administration (FDA)-approved tagraxofusp, a novel agent designed to target the mechanism of BPDCN’s action.

MD Mag: What were the findings of your team's study of tagraxofusp in patients with blastic plasmacytoid dendritic-cell neoplasm?

Pemmaraju: I was honored to lead a team of investigators to have a major paper recently published in the New England Journal of Medicine, which was on a novel agent known as tagraxofusp, or SL-401—phase 1 and 2 results for a rare but highly aggressive tumor known as BPDCN: blastic plasmacytoid dendritic-cell neoplasm.

This paper represented not only our first FDA-approved drug in our field for a targeted agent in BPDCN, but also more importantly, the first-ever CD-123 specifically-targeting agent in any area of oncology.

Briefly, the background and results were that we treated 45 patients with BPDCN, and we showed that an approximately 90% overall response rate in the frontline setting, and a 60-plus percent response rate in the relapse-refractory setting.

Among these 45 patients, one of the important items that we noted were that the majority of these responses were complete remissions, and the safety profile showed a very important signal, which is called capillary leak syndrome, which is a fluid retention syndrome.

This appropriately has a Black Box warning in the package insert, as we have seen in other drug approvals before. So overall, the FDA approved this drug in December 2018. The New England Journal paper is now published with exclusive details of the experience, and this is an exciting move forward and may represent a very new standard of care for many of our patients with BPDCN.