TransCon CNP is navepegritide, marketed as YUVIWEL, a once-weekly C-type natriuretic peptide analog approved to increase linear growth in pediatric patients aged 2 years and older with achondroplasia and open epiphyses. The indication is under accelerated approval with confirmatory benefit follow-up required. U.S. initial approval was in 2026.
The FDA has granted accelerated approval for YUVIWEL (navepegritide), the first once-weekly treatment for children with achondroplasia. Commercial availability is expected in early Q2 2026, with a Rare Pediatric Disease Priority Review Voucher granted.
BridgeBio Pharma reported positive Phase 3 results for oral infigratinib in children with achondroplasia, meeting primary and secondary endpoints with superior growth velocity and the first statistically significant improvement in body proportionality. The company plans regulatory submissions in the second half of 2026.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT05929807 |
A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia |
ENROLLING_BY_INVITATION | PHASE2/PHASE3 |
| NCT05598320 |
A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children With Achondroplasia |
COMPLETED | PHASE2/PHASE3 |
| NCT05246033 |
A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Multiple Subcutaneous Doses of TransCon CNP Administered Once Weekly in Children With Achondroplasia |
ACTIVE_NOT_RECRUITING | PHASE2 |
| NCT04085523 |
A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia |
COMPLETED | PHASE2 |
