FDA granted fast track and rare pediatric disease designations to WU-CART-007 for R/R T-ALL/LBL

The ALL Hub has previously reported on the granted orphan drug designation for WU-CART-007 by the U.S. Food and Drug Administration (FDA) for relapsed/refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL). Further to this clinical development, on July 19, 2022, WU-CART-007 received both fast track and rare pediatric disease designations by the FDA for the treatment of R/R T-ALL/lymphoblastic lymphoma.¹

The phase I/II global, open-label, first-in-human trial of WU-CART-007 is currently ongoing to evaluate the safety and efficacy in an estimated 44 patients with R/R T-ALL/lymphoblastic lymphoma. The study consists of two phases 1) phase I dose escalation and 2) phase II expansion phase. The primary endpoints are response rates and safety including adverse effects, maximum tolerated dose, overall response rate, duration of response, and progression-free survival. The secondary endpoints include overall survival and hematopoietic stem cell transplant rate. If proven effective and safe in these trials, WU-CART-007 could offer a new treatment option and address an important unmet need in these populations. The study is expected to be completed in August 2026.²