Navepegritide

The labelled product is Yuviwel (Ascendis; aka TransCon CNP). FDA granted accelerated approval (content current as of 4 Mar 2026 on the FDA notice; NDA 219164 approval package dated 27 Feb 2026) to increase linear growth in pediatric patients 2 years and older with achondroplasia and open epiphyses. Approval is based on annualized growth velocity; a confirmatory final-adult-height study is required. Another labelled CNP-pathway medicine for achondroplasia is vosoritide (Voxzogo); that is a separate product and is not drafted here. This is not a general “height peptide.” Research-chemical CNP listings are not Yuviwel.

A C-type natriuretic peptide (CNP) analog prodrug. Released CNP binds NPR-B, increases cGMP signaling, and antagonizes overactive FGFR3/MAPK signaling that impairs endochondral ossification in achondroplasia. Labelled intent is increased linear growth in the indicated pediatric population with open epiphyses—not a general height or anti-aging peptide.

APPROACH / Trial 1 (NCT05598320): randomized, double-blind, placebo-controlled 52-week period in 84 treatment-naïve children with genetically confirmed achondroplasia (navepegritide n=57; placebo n=27), published as Savarirayan et al., JAMA Pediatrics 2026 (PMID 41247754). FDA notice and Yuviwel label report least-squares mean AGV treatment difference of 1.5 cm/year at Week 52 (5.9 vs 4.4 cm/year). Height Z-score changes were also reported. Open-label extension data describe maintained AGV with continued treatment. Accelerated approval requires confirmatory evidence on final adult height. That is why the grade is Moderate, not High.

• FDA approval notice — Yuviwel for pediatric achondroplasia · 2026

  FDA approves drug for pediatric patients with most common form of dwarfism. Yuviwel (navepegritide) for ages ≥2 with achondroplasia and open epiphyses. Accelerated approval on AGV (~1.5 cm vs placebo over 52 weeks). Confirmatory final adult height study required. Content current as of 03/04/2026.

• Yuviwel (navepegritide) DailyMed label · 2026

  Prescribing information. CNP analog; accelerated approval on AGV. NDA 219164. Setid e01c1197-338f-414c-b804-808e129ebc8c. Transient hypotension class warning. §4 contraindications: none.

• Savarirayan et al., APPROACH · 2026 · JAMA Pediatr

  Once-Weekly Navepegritide in Children With Achondroplasia: The APPROACH Randomized Clinical Trial. JAMA Pediatr. 2026. PMID 41247754. DOI 10.1001/jamapediatrics.2025.4771. NCT05598320. PMID 41247754.

• APPROACH ClinicalTrials.gov · 2022

  NCT05598320. A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children With Achondroplasia. Phase 2/3; completed.

• FDA NDA 219164 approval package · 2026

  Approval package PDF for Yuviwel (navepegritide), NDA 219164Orig1s000. Approval date February 27, 2026. Indication: increase linear growth in pediatric patients ≥2 with achondroplasia with open epiphyses.