FDA

FDA grants accelerated approval to Yuviwel for achondroplasia in children

March 4, 2026

Brand name: Yuviwel

Generic name: navepegritide

Manufacturer: Ascendis Pharma

Approval date: February 27, 2026

Yuviwel (navepegritide), a once-weekly SC injection, was approved to increase linear growth in pediatric patients 2 years of age and older with achondroplasia with open epiphyses. Navepegritide is a prodrug of C‑type natriuretic peptide (CNP) that provides steady CNP exposure throughout the weekly dosing interval, helping to counter the overactive fibroblast growth factor receptor 3 (FGFR3) signaling seen in achondroplasia.

Efficacy

Yuviwel's accelerated approval was supported by three randomized, double-blind, placebo-controlled trials, and up to 3 years of open-label extension data. The pivotal phase 3 ApproaCH trial showed that Yuviwel was superior to placebo for the primary endpoint—annualized growth velocity (AGV) at week 52. Improvement in AGV was a surrogate marker reasonably likely to predict clinical benefit, however, continued approval of this indication is contingent upon confirmatory studies to verify longer‑term benefits.

Significant improvements in additional measures, such as tibial‑femoral angle, mechanical axis deviation, fibula‑to‑tibia length ratio, and scores on the Achondroplasia Child Experience Measures-Physical Functioning, were also observed in the ApproaCH study.

Safety

Most common adverse reactions (≥ 5% and greater than placebo): vomiting, injection-site reactions, pain in extremity, and nausea. Less common was hypertrichosis (3%).

Yuviwel has a warning for low blood pressure risk, as transient decreases in blood pressure have been reported with a once-daily CNP analog.

Sources:

Ascendis Pharma. (2026, February 27). FDA Approves Once-Weekly Yuviwel (navepegritide) for Children with Achondroplasia Aged 2 Years and Older. [Press release]. https://investors.ascendispharma.com/news-releases/news-release-details/fda-approves-once-weekly-yuviwelr-navepegritide-children

Yuviwel (navepegritide). [Package insert]. Ascendis Pharma. https://ascendispharma.us/products/pi/yuviwel/yuviwel_pi.pdf Revised February 2026. Accessed March 3, 2026.

Savarirayan R, et al. (2025, November 17). JAMA Pediatr. Once-Weekly Navepegritide in Children With Achondroplasia: The APPROACH Randomized Clinical Trial. https://jamanetwork.com/journals/jamapediatrics/fullarticle/2841492