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 primarily affects bone growth in childhood, resulting in short arms and legs and an average adult height under 4 feet 4 inches. Yuviwel is a long-acting, once-weekly prodrug that delivers a protein called C-type natriuretic peptide (CNP) to the body. By mimicking this natural growth signal, the treatment may help increase linear growth in children whose bones are still developing.
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