CHMP recommends EU marketing authorisation of Yuviwel (navepegritide) for achondroplasia in children aged 2 years and older
The European Medicines Agency's human medicines committee (CHMP) adopted a positive opinion on October 15, 2026 recommending marketing authorisation for Yuviwel (navepegritide, Ascendis Pharma), a prodrug of C-type natriuretic peptide, for the treatment of achondroplasia in children aged 2 years and older whose growth plates are still open. The opinion is a recommendation, not an approval: a European Commission decision is pending.
The Committee for Medicinal Products for Human Use adopted its positive opinion on October 15, 2026, and the European Medicines Agency published it on October 16. Yuviwel (navepegritide), developed by Ascendis Pharma Growth Disorders A/S, is a prodrug of C-type natriuretic peptide (CNP). It acts as an agonist of the NPR-B receptor, promoting the proliferation and differentiation of chondrocytes, the cells that drive bone growth. It is indicated for the treatment of achondroplasia in patients aged 2 years and older whose epiphyses (growth plates) are not closed; the diagnosis must be genetically confirmed, and treatment should be initiated and supervised by a physician experienced in the management of growth disorders.
The recommendation is supported by a randomised, double-blind, placebo-controlled trial of 52 weeks' duration in which navepegritide improved annualised growth velocity by 1.49 cm relative to placebo. The medicine is supplied as a powder and solvent for solution for injection in 1.3 mg, 2.8 mg and 5.5 mg strengths.
The most common side effects are injection-site reactions and hypertrichosis (excessive hair growth). Yuviwel was designated an orphan medicine, and the orphan status is under maintenance review at the time of the opinion. A CHMP positive opinion is not a marketing authorisation: the European Commission must still adopt a legally binding decision before Yuviwel can be marketed in the European Union (application number EMEA/H/C/006627).