The U.S. Food and Drug Administration approved tagraxofusp-erzs for the treatment of adults and children (≥2 years of age) with blastic plasmacytoid dendritic cell neoplasm (BPDCN), marking the first approval for this rare disease.
The efficacy of tagraxofusp-erzs was evaluated in two cohorts of patients in a single-arm clinical trial. In the first cohort, which included 13 patients with untreated BPDCN, seven (54%) achieved complete remission (CR) or CR with a skin abnormality not indicative of active disease (CRc); in the second cohort, which included 15 patients with relapsed or refractory BPDCN, one patient each achieved CR and CRc.
Common adverse events associated with tagraxofusp-erzs included capillary leak syndrome, nausea, fatigue, peripheral edema, pyrexia, chills, and weight increase. The most commonly observed laboratory abnormalities were decreases in lymphocytes, albumin, platelets, hemoglobin and calcium, and increases in glucose and liver enzymes. Given this observation, the approval letter advises health-care providers to monitor patients’ liver enzyme levels.
Tagraxofusp-erzs was approved with a boxed warning about the increased risk of capillary leak syndrome, which may be life-threatening or fatal.
This agent also was granted breakthrough therapy, priority review, and orphan drug designations.
Source: FDA news release, December 21, 2018.