Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial
This Phase III study (n=121) found once-daily subcutaneous vosoritide was associated with increased growth versus placebo in children with achondroplasia (adjusted mean difference in annualised growth velocity of 1.57cm/year in favour of vosoritide; 95% CI 1.22–1.93]; p<0.0001).
Source:
The Lancet
SPS commentary:
Achondroplasia, the most common form of disproportionate short stature in humans, is a primary skeletal dysplasia caused by heterozygous, gain-of-function mutations in the fibroblast growth factor receptor 3 (FGFR3) gene that leads to impaired endochondral ossification. Vosoritide is a C-type natriuretic peptide stimulant that initiates intracellular signals to inhibit the overactive FGFR3 pathway and normalise bone growth. An application for its approval in the EU was filed in July 2020.
The researchers note that there were no adverse effects on, or significant improvements in upper to lower body segment proportionality in children receiving vosoritide during this 52 week study, suggesting that either a longer treatment period or earlier treatment initiation might be required to detect these changes. It is not known whether treatment will increase final adult height, and whether it will ameliorate the medical complications associated with achondroplasia and decrease the need for surgical interventions.