CHMP backs weekly navepegritide for achondroplasia

EU regulators back weekly navepegritide for children with achondroplasia

Europe's medicines committee has recommended Ascendis Pharma's once-weekly navepegritide for children with achondroplasia. If the European Commission agrees, it will compete with BioMarin's daily vosoritide.

By NextGenHealth Editorial Team4 min read

In brief

The CHMP has recommended EU approval of Ascendis Pharma's once-weekly navepegritide for children aged two and over with achondroplasia.

The CHMP adopted a positive opinion on navepegritide (Yuviwel) for achondroplasia in children aged two and over at its 12 to 15 October 2026 meeting.

In the pivotal ApproaCH trial, children on the weekly drug grew 1.49 cm a year faster than those on placebo after 52 weeks.

The FDA granted accelerated approval in February 2026; a European Commission decision is expected in the fourth quarter of 2026.

The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recommended approval of navepegritide, Ascendis Pharma's once-weekly treatment for achondroplasia, the most common form of dwarfism. The opinion was adopted at the committee's meeting of 12 to 15 October 2026 and announced by both the agency and the company on 16 October.

The recommended indication is the treatment of achondroplasia in children two years of age and older whose epiphyses, or growth plates, are not closed. The drug will be sold as Yuviwel, the name already used in the US. It is an orphan medicine, and the EMA said it will review whether the orphan designation can be maintained.

How the drug works

Achondroplasia is caused by an overactive fibroblast growth factor receptor 3 (FGFR3) gene, which restrains bone growth. Navepegritide is a C-type natriuretic peptide (CNP) analogue, a class designed to counter that signalling. It is built on Ascendis's TransCon platform, which allows a once-weekly subcutaneous injection.

BioMarin's vosoritide (Voxzogo), another CNP analogue, has held EU marketing authorisation since August 2021. According to its EMA product page, it is given as a daily injection and is now indicated from four months of age.

The evidence

Ascendis said its EU application included data from three randomised, double-blind, placebo-controlled trials, with up to three years of open-label extension data. The pivotal study, ApproaCH, was published in JAMA Pediatrics.

ApproaCH enrolled 84 treatment-naive children aged two to 11 with genetically confirmed achondroplasia. They were randomised 2:1 to navepegritide 100 micrograms per kilogram a week or placebo for 52 weeks, followed by an open-label extension. At week 52, annualised growth velocity was 5.89 cm a year on the drug against 4.41 cm on placebo, a difference of 1.49 cm a year (95% confidence interval 1.05 to 1.93; p<0.001), as reported by Pharmacy Times. The effect was smaller in children under five (1.02 cm a year) than in those aged five and over (1.78 cm a year).

Ascendis also reported improvements in body proportions and in measures of leg alignment, without acceleration of bone age. The company said the safety profile was similar to placebo, with most adverse events mild or moderate and no symptomatic hypotension or fractures reported. The most common adverse events on navepegritide included fever, nasopharyngitis and ear infections. Injection-site reactions occurred in 19.3% of treated children against 14.8% on placebo.

The US precedent

The FDA granted accelerated approval to Yuviwel on 27 February 2026 for children aged two and over with achondroplasia and open growth plates. The agency based its decision on annualised growth velocity, a surrogate endpoint, and said continued approval depends on an ongoing study confirming an improvement in final adult height. Ascendis said the FDA also issued it a rare paediatric disease priority review voucher.

That question of final height, and of whether faster growth in childhood translates into fewer of the medical complications of achondroplasia, applies to the whole drug class. It will take years of follow-up to answer.

What comes next

The CHMP opinion now goes to the European Commission, which Ascendis expects to decide in the fourth quarter of 2026. The company plans its first EU launch, in Germany, in the first quarter of 2027. "We are pleased to receive this positive opinion reflecting the unique profile of TransCon CNP," said Jan Mikkelsen, Ascendis's president and chief executive.

For payers, the arrival of a second CNP analogue raises the prospect of competition in a small market. For families, the practical difference is the dosing schedule: one injection a week rather than seven. Whether that convenience changes uptake or adherence has not yet been tested in a head-to-head trial.