The US FDA has approved Tagraxofusp infusion for the treatment of adults and pediatric patients, age ≥ 2 years, with Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN). The FDA has granted the drug with breakthrough therapy, priority review, and orphan drug designations. Tagraxofusp is directed at the Interleukin-3 receptor, which is overexpressed on cancer stem cells or tumor bulk in several hematologic malignancies. The approval was based on a clinical trial in patients with untreated or relapsed/refractory BPDCN.
Patients received the drug at the recommended dose of 12 mcg/kg. In the first cohort, 7 out of 13 patients with untreated BPDCN achieved complete response/clinical complete response after a median follow-up of 11.5 months. In the second cohort of 15 patients, one patient achieved a complete response within 111 days and one patient achieved a clinical complete response…
