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lonapegsomatropin
- Development stage
- Approved
- Lead developer
- Ascendis Pharma
- Modality
- Recombinant Proteins and Enzymes, Metabolically Activated Prodrugs → Prodrugs/Conditional Activator Small Molecules → Small Molecules
- Administration
- Subcutaneous
01
Overview
Lonapegsomatropin is a long-acting prodrug of human growth hormone (somatropin) designed for once-weekly subcutaneous administration to treat pediatric patients with growth failure due to inadequate endogenous growth hormone secretion. It consists of unmodified recombinant human growth hormone covalently attached to a methoxypolyethylene glycol (mPEG) carrier via a transient linker, which undergoes autohydrolysis in the body, allowing controlled release of native somatropin over one week. The released somatropin binds to the human growth hormone receptor, activating direct and insulin-like growth factor 1 (IGF-1)-mediated effects that promote linear bone growth and regulate metabolism. Lonapegsomatropin has demonstrated noninferiority and even superiority in annualized height velocity compared to daily somatropin injections in clinical trials. It was developed by Ascendis Pharma and is marketed under the brand name Skytrofa[1][3][6][8].
- Brand names
- Skytrofa
- Other names
- lonapegsomatropin-tcgd
02
Targets
GHR (Growth hormone receptor)
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