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 subcutaneous vosoritide, while 61 received placebo injections.

All had taken part in a six-month study of their growth prior to starting the trial, wrote the study authors, led by Professor Ravi Savarirayan of the Murdoch Children’s Research Institute, Royal Children’s Hospital and University of Melbourne.