On January 7, 2021, the China National Medical Products Administration (NMPA) Center for Drug Evaluation (CDE) granted the E-selectin inhibitor APL-106 breakthrough therapy designation for the treatment of patients with relapsed/refractory (R/R) acute myeloid leukemia (AML).
The agent received investigational new drug approval in September 2020, which provided grounds for a future phase III bridging study of APL-106 in combination with chemotherapy for the treatment of R/R AML.1
APL-106 is a first-in-class, investigational, targeted inhibitor of the E-selectin adhesion molecule.1 E-selectin is expressed in the bone marrow endothelium and is essential for physiological hematopoietic turnover. Upon interaction with AML cells, however, E-selectin can promote survival and regeneration, and is now a recognized contributor to AML chemoresistance.2
This newly introduced designation is designed to hasten the development and health authority review of APL-106 in R/R AML. Further research will demonstrate whether this new drug provides an advantage over existing treatment options.