VOXZOGO
VOXZOGO (vosoritide) is a once-daily subcutaneous injection developed by BioMarin for achondroplasia — the most common form of short-limb dwarfism, caused by a gain-of-function FGFR3 mutation that suppresses bone growth at the growth plates. The drug is a synthetic analog of C-type natriuretic peptide (CNP), a signaling molecule that inhibits the overactive FGFR3 receptor, allowing growth plates to develop more normally and increasing annualized height velocity in children. VOXZOGO is approved in the US for patients aged 5 and older; BioMarin is pursuing a label extension for infants from age 4 months.
Upcoming catalysts
Programs
Achondroplasia
Achondroplasia — the most common skeletal dysplasia at 1 in 25,000 births — is caused by a dominant FGFR3 gain-of-function mutation suppressing bone growth at the growth plates, resulting in short stature and orthopedic complications. VOXZOGO is approved in the US for patients aged 5+; this sNDA program targets label extension to infants aged 4 months to under 5, supported by Phase 2 data showing +1.10 cm/yr greater annualized height velocity versus placebo and a November 2026 PDUFA.