The U.S. Food and Drug Administration (FDA) has approved the drug Yuviwel (navepegritide) from the global biopharmaceutical company Ascendis Pharma for the treatment of children with achondroplasia, the most common form of genetic dwarfism. The medication is intended for patients aged two years and older.
Yuviwel has become the first weekly-administered drug that neutralizes the overactivity of the FGFR3 receptor. Its mechanism of action is based on the prolonged release of C-type natriuretic peptide (CNP), which stimulates linear growth.
The approval was granted under an accelerated procedure based on data from the 52-week ApproaCH Trial. The study in children aged 2–11 years recorded a statistically significant increase in the annualized growth rate compared to placebo. Specifically, children receiving Yuviwel achieved an annualized growth velocity of 5.89 cm compared to 4.41 cm in the placebo group. Ascendis Pharma plans to launch the drug in the US market in early 2026.
Yuviwel will compete with the drug Voxzogo (vosoritide) from the biotechnology company BioMarin, which requires daily injections and is insufficiently effective in about 20% of patients, according to Reuters. Analysts forecast peak sales for Yuviwel at €772 million by 2032. The success of BridgeBio’s developments with an oral drug confirms the growing interest in therapies aimed at correcting FGFR3 mutation.
Achondroplasia is the most common type of short-limbed dwarfism. According to the National Institutes of Health, this condition occurs in 1 in 15,000 to 40,000 newborns.
