BioMarin Pharmaceutical has won National Medical Products Administration (NMPA) approval in China for vosoritide, a C-type natriuretic peptide analogue and the first disease-modifying therapy for achondroplasia, marking China's latest greenlight for a Western-developed rare-paediatric medicine after earlier EU and US approvals in 2021 for children aged 5 years and older with open growth plates.

Achondroplasia is driven by a gain-of-function FGFR3 mutation that negatively regulates endochondral bone growth, and vosoritide acts downstream as a positive modulator of the FGFR3 pathway to promote cartilage-to-bone formation, rather than merely managing orthopaedic or neurological complications.

The registration case rested on a randomised, double-blind, placebo-controlled Phase III study in patients aged 5 years and older, dosed once daily for 52 weeks, where vosoritide lifted annualised growth velocity by 1.57 cm/year versus placebo, a modest but clinically meaningful shift for a condition with no previously approved pharmacological growth treatment.