Navepegritide (TransCon CNP)
Navepegritide (TransCon CNP) is a long-acting prodrug of C-type natriuretic peptide, or CNP, in development for the treatment of achondroplasia, or ACH, in children, which is the first achondroplasia disease-modifying therapy in clinical development in China to date. It is designed to provide continuous CNP exposure which inhibits the effect of constitutive fibroblast growth factor receptor 3 (FGFR3) over-activation 24 hours a day, aiming to promote bone growth and ameliorate and prevent complications and comorbidities of ACH.
The China Phase 2 trial of navepegritide for achondroplasia (ACH) was successfully completed and met its primary endpoint. Navepegritide (TransCon CNP) was granted orphan drug recognition for the treatment of achondroplasia by U.S. Food & Drug Administration (FDA) in February 2019 and European Commission (EC) in August 2020.