CHMP Backs Once-Weekly TransCon CNP for Children With Achondroplasia – Ascendis Pharma
Ascendis Pharma announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending marketing authorization for once-weekly TransCon CNP (navepegritide) in the European Union for the treatment of achondroplasia in children two years of age and older whose epiphyses are not closed.
The final European Commission (EC) decision on Ascendis Pharma’s Marketing Authorisation Application (MAA) is expected during Q4 2026.
“We are pleased to receive this positive opinion reflecting the unique profile of TransCon CNP,” said Jan Mikkelsen, President and Chief Executive Officer at Ascendis Pharma. “With a highly favorable response from the achondroplasia community to its differentiated profile, TransCon CNP remains a foundational product in our growth disorder portfolio, positioned to support our Vision 2030 objectives.”
CHMP adopted its positive opinion following its review of the MAA for TransCon CNP, which included clinical data from three randomized, double-blind, placebo-controlled clinical trials and up to three years of open-label extension data. The pivotal ApproaCH Trial data is available in JAMA Pediatrics.
TransCon CNP was designated as an orphan medicine during its development. EMA will now review the information available to date to determine if the orphan designation can be maintained.
If authorized by the EC, Ascendis expects its first EU launch of TransCon CNP under the brand name YUVIWEL to be in Germany during Q1 2027.
Citation: Savarirayan R, McDonnell C, Bacino CA et al. Once-Weekly Navepegritide in Children With Achondroplasia: The APPROACH Randomized Clinical Trial. JAMA Pediatr. Published online November 17, 2025. doi:10.1001/jamapediatrics.2025.4771
