First targeted therapy for achondroplasia effective: trial

Australian children took part in the trial that could fundamentally change management, authors say

Children with achondroplasia receiving weekly vosoritide injections have a greater annualised growth rate than those given placebo, a world-first study shows.


The international trial across 24 sites and seven countries, including Australia, was initiated and run by BioMarin Pharmaceutical, the manufacturer of the therapy, a biological analogue of C-natriuretic peptide.

Some 60 children aged between five and 18 (mean age 8.7 years) were randomised to a year of weekly 15mcg/kg