On December 21, the U.S. Food and Drug Administration (FDA) approved tagraxofusp-erzs (Elzonris) infusion for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and in pediatric patients aged 2 years and older.
“Prior to [this] approval, there had been no FDA-approved therapies for BPDCN. The standard of care has been intensive chemotherapy followed by bone marrow transplantation. Many patients with BPDCN are unable to tolerate this intensive therapy, so there is an urgent need for alternative treatment options,” said Richard Pazdur, MD, Director of the FDA’s Oncology Center of Excellence and Acting Director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research.
BPDCN is an aggressive and rare disease of the bone marrow and blood that can affect multiple organs, including the lymph nodes and the skin. It often presents as leukemia or evolves into acute leukemia. The disease is more common in men than women and in patients 60 years and older.
Approval was based on a multicenter, multicohort, open-label, single-arm clinical trial (STML-401-0114) in patients with untreated or relapsed/refractory BPDCN. Patients received tagraxofusp-erzs at the recommended dose of 12 μg/kg.
In the pivotal cohort, 7 (53.8%; 95% confidence interval [CI] = 25.1%–80.8%) of 13 patients with untreated BPDCN achieved a complete response or clinical complete response after a median follow-up of 11.5 months. The median response duration was not reached. In the second cohort, of 15 patients with relapsed or refractory BPDCN, 1 patient achieved a complete response (duration = 111 days) and 1 patient achieved a clinical complete response (duration = 424 days).
The most common adverse reactions (incidence ≥ 30%) reported were capillary leak syndrome, nausea, fatigue, peripheral edema, pyrexia, and weight increase. The most common laboratory abnormalities (incidence ≥ 50%) were decreases in albumin, platelets, hemoglobin, calcium, and sodium; and increases in glucose, alanine aminotransferase, and aspartate aminotransferase.
Health-care providers are advised to monitor liver enzyme levels and for signs of intolerance to the infusion. The labeling for tagraxofusp-erzs contains a Boxed Warning to alert health-care professionals and patients about the increased risk of capillary leak syndrome, which may be life-threatening or fatal to patients in treatment.
The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.