The U.S. FDA granted accelerated approval to YUVIWEL (navepegritide; TransCon CNP), the first once-weekly injection for children aged 2 years and older with achondroplasia and open epiphyses, based on improved annualized growth velocity.123
Approval occurred around February 27, 2026, with commercial availability expected in the early part of Q2 2026 through prescribing physicians.124
YUVIWEL is a prodrug of C-type natriuretic peptide (CNP) providing continuous systemic exposure to support bone growth by dampening overactive FGFR3 signaling.12
The approval is subject to confirmatory trials to verify long-term clinical benefits; common side effects include injection-site reactions and low blood pressure.23
FDA also issued Ascendis a Rare Pediatric Disease Priority Review Voucher.12
YUVIWEL competes with BioMarin's daily Voxzogo, potentially capturing patients preferring weekly dosing; analysts project modest 2026 sales with growth in 2027.3
Sources:
1. https://www.stocktitan.net/sec-filings/ASND/6-k-ascendis-pharma-a-s-current-report-foreign-issuer-1374695a817c.html
2. https://globalgenes.org/raredaily/fda-grants-ascendis-accelerated-approval-for-achondroplasia-drug/
3. https://www.biopharmadive.com/news/ascendis-fda-approval-yuviwel-achondroplasia-biomarin-voxzogo/813502/