BioMarin Pharmaceutical has submitted vosoritide for Chinese approval as the first therapy for paediatric achondroplasia, following its 2021 accelerated approval in the US for children aged five years and older with open growth plates. The C-type natriuretic peptide analogue counteracts FGFR3 mutations that impair endochondral bone growth, demonstrating 1.57 cm/year greater linear growth versus placebo in Phase III trials. The treatment addresses unmet needs in dwarfism where no pharmacological interventions previously existed.

Vosoritide is approved in the European Union, Japan, South Korea and the US, with additional development ongoing for Turner syndrome. BioMarin's submission to China's Centre for Drug Evaluation (CDE) expands access to this breakthrough therapy in a market where achondroplasia affects approximately 50,000 patients. The daily subcutaneous injection represents a novel approach to skeletal dysplasia management beyond traditional growth hormone therapies.

PharmCube's NextBiopharm® database shows that vosoritide is one of only three achondroplasia therapies approved globally, all of which got registered in 2021. Click here to request a free trial for NextBiopharm®.