The US Food and Drug Administration (FDA) has granted accelerated approval of Yuviwel (navepegritide; previously TransCon CNP) for the treatment of patients two years of age and older with achondroplasia.
Achondroplasia is a disorder of bone growth that prevents the changing of cartilage (particularly in the long bones of the arms and legs) to bone. It is characterized by dwarfism, disproportionate short stature, macrocephaly, and unique facial/spinal features. Achondroplasia can cause health complications such as apnea, obesity, recurrent ear infections, and lordosis. More serious problems include spinal stenosis and hydrocephalus. Some people with achondroplasia may have delayed motor development early on, but cognition is normal. It is caused by genetic changes in the FGFR3 gene.
Navepegritide is a prodrug of C-type natriuretic peptide (CNP), administered once weekly, and designed to provide continuous exposure of active CNP to postively impact bone growth. The treatment is indicated for children ages two years of age and older with achondroplasia with open epiphyses and the only one to provide continuous systemic exposure to CNP over the weekly dosing interval
The approval is based on the FDA’s review of the clinical package for TransCon CNP submitted with the New Drug Applications, which included safety and efficacy data from three randomized, double-blind, placebo-controlled clinical trials and up to three years of open-label extension data.
The approval was largely based on the ApproaCH clinical trial where navepegritide demonstrated superiority in annualized growth velocity at week 52 compared to placebo. Improvements were also observed in tibial-femoral angle, mechanical axis deviation, fibula to tibia length ratio, and Achondroplasia Child Experience Measures-Physical Functioning. Full results from the ApproaCH study can be found in JAMA Pediatrics.
Navepegritide is expected to be available in the second quarter of 2026. With this approval, the FDA also issued a Rare Pediatric Disease Priority Review Voucher. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
For more information, visit the Press Release.
To learn more about achondroplasia and other rare genetic conditions, visit https://checkrare.com/diseases/congenital-and-genetic-conditions/