for achondroplasia in children aged 2 years and older with open epiphyses
FDA granted accelerated approval to Yuviwel (navepegritide) on February 27, 2026 — three days ahead of the extended Mar 2 2026 PDUFA target. First and only once-weekly treatment indicated to increase linear growth in children aged 2 yrs and older with achondroplasia (open epiphyses). Rare Pediatric Disease Priority Review Voucher issued.
Achondroplasia is a genetic disorder that disrupts bone growth, making arms and legs short while the torso stays near normal length, and it can bring complications such as sleep apnea or repeated ear infections. Yuviwel is a once-weekly injection of a lab-made version of C-type natriuretic peptide, a natural hormone that binds receptors in growing bone and calms the signals that normally stop cartilage cells from dividing. Because that braking signal is overactive in achondroplasia, delivering extra CNP may help children's bones keep lengthening during the years when their growth plates are still open.
The purpose of this clinical trial was to evaluate efficacy and safety of once weekly subcutaneous (SC) doses of 100 µg TransCon CNP/kg compared to placebo on Annualized Growth Velocity after a 52-week randomized treatment period in children aged 2 to 11 years with genetically confirmed Achondroplasia. The double-blind, placebo-controlled treatment period was followed by an Open Label Extension (OLE) period of a 52-week duration.
Source: ClinicalTrials.gov