The US Food and Drug Administration has granted accelerated approval to navepegritide (YUVIWEL) to increase linear growth in pediatric patients aged 2 years and older with achondroplasia and open epiphyses, based on improvements in annualized growth velocity observed in randomized clinical trials.
Approval was based on improvements in annualized growth velocity observed in three randomized, double-blind, placebo-controlled clinical trials, supported by up to 3 years of open-label extension data. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials. Findings from the pivotal ApproaCH trial were published in JAMA Pediatrics.
Achondroplasia is a rare genetic condition arising from a systemic fibroblast growth factor receptor 3 variant and historically characterized as a skeletal dysplasia. The receptor variant is expressed in tissues throughout the body and is associated with increased risk of muscular, neurological, and cardiorespiratory complications. In pediatric patients, reported complications include spinal abnormalities, ventriculomegaly, reduced muscle strength and stamina, hearing impairment, upper airway obstruction, sleep-disordered breathing, hip abnormalities, leg bowing, and chronic pain. Some patients undergo multiple surgical interventions to address structural or functional concerns.
Navepegritide is a prodrug of C-type natriuretic peptide designed to provide continuous systemic exposure to active peptide over a weekly dosing interval, thereby counteracting overactive fibroblast growth factor receptor 3 signaling.
The US Food and Drug Administration also issued a Rare Pediatric Disease Priority Review Voucher in connection with the approval, a mechanism intended to incentivize development of therapies for rare pediatric conditions.
Serious adverse reactions may include hypotension. Caregivers should seek medical evaluation if symptoms consistent with low blood pressure, including dizziness, fatigue, or nausea, occur. The most reported adverse events were injection site reactions, including erythema, pruritus, discoloration, bleeding, swelling, bruising, pain, and blistering.
Commercial availability in the US is anticipated in early Q2 2026.
Source: Ascendis Pharma
