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lonapegsomatropin + navepegritide
- Development stage
- Unknown
- Lead developer
- Ascendis Pharma
- Modality
- Peptides, Recombinant Proteins and Enzymes
- Administration
- Subcutaneous
01
Overview
Lonapegsomatropin + navepegritide is an investigational combination therapy developed by Ascendis Pharma for the treatment of achondroplasia in children. The regimen consists of two once-weekly subcutaneous injections: navepegritide (TransCon CNP), a prodrug of C-type natriuretic peptide (CNP), and lonapegsomatropin (TransCon hGH), a prodrug of human growth hormone (somatropin). Navepegritide targets the overactive fibroblast growth factor receptor 3 (FGFR3) pathway by activating CNP receptors, while lonapegsomatropin promotes linear growth through the growth hormone/IGF-1 axis. This dual-agent approach is designed to provide complementary effects on growth plates and skeletal development, aiming to improve annualized growth velocity and body proportionality. Clinical data from the Phase 2 COACH trial demonstrated significant improvements in growth velocity and proportionality compared to monotherapy.
- Brand names
- Skytrofa
- Other names
- TransCon hGH + TransCon CNP
02
Targets
GHR (Growth hormone receptor)NPR2 (C-type Natriuretic Peptide Receptor B)
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