Carlos A. Bacino, MD, Professor of Molecular and Human Genetics, Baylor College of Medicine and Texas Children’s Hospital, discusses the accelerated approval of Yuviwel (navepegritide) for patients with achondroplasia.
Achondroplasia is a disorder of bone growth that prevents the changing of cartilage to bone, particularly in the long bones of the arms and legs. It is characterized by dwarfism, limited range of motion at the elbows, macrocephaly, small fingers, and normal intelligence. Achondroplasia can cause health complications such as apnea, obesity, recurrent ear infections, and lordosis. More serious problems include spinal stenosis and hydrocephalus. Some patients may have delayed motor development early on, but cognition is normal. Achondroplasia is caused by genetic changes in the FGFR3 gene.
The US Food and Drug Administration (FDA) recently granted Accelerated Approval to navepegritide for the treatment of children 2 years of age and older with achondroplasia. As Dr. Bacino explains, this is the first and only once-weekly treatment and it is indicated to increase linear growth in patients with open epiphyses and the only treatment to provide continuous systemic exposure to C-type natriuretic peptide (CNP) over the weekly dosing interval.
Navepegritide is a prodrug of CNP administered once weekly, designed to provide continuous exposure of active CNP to receptors on tissues throughout the body to counteract the overactive FGFR3 signaling in achondroplasia.
The approval was largely based on the safety and efficacy data from three randomized, double-blind, placebo-controlled clinical trials and up to three years of open-label extension data. The pivotal ApproaCH Trial data is available in JAMA Pediatrics.
Navepegritide is expected to be available in the US in the second quarter of 2026. Continued approval for this indication, which was based on an improvement in annualized growth velocity (AGV), may be contingent upon verification and description of clinical benefit in confirmatory trials.
Alongside this approval, the FDA also issued a Rare Pediatric Disease Priority Review Voucher (PRV), which confers priority review to a subsequent drug application that would not otherwise qualify for priority review. This program is designed to encourage development of new drugs and biologics for the prevention or treatment of rare pediatric diseases.
To learn more about the FDA approval, visit FDA Grants Accelerated Approval to Navepegritide for the Treatment of Patients With Achondroplasia.
To learn more about the value of recent PRVs, visit checkrare.com/priority-review-voucher/