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 causing dwarfism, where shortened limbs and spinal complications lead to an average adult height under 4'4", often accompanied by breathing difficulties and recurrent infections. Yuviwel delivers a long-acting version of CNP, a natural growth-regulating protein, which works by stimulating bone growth plates to counteract the genetic slowdown in skeletal development. This weekly injection may help children with achondroplasia achieve greater height potential while avoiding the need for daily treatments.
The trial is a multicenter, double-blind, randomized, placebo-controlled, dose escalation trial of weekly TransCon CNP administered subcutaneously in prepubertal children 2 to 10 years old, inclusive, with Achondroplasia.
Source: ClinicalTrials.gov