Achondroplasia therapy options widen past vosoritide

Achondroplasia has been a supportive-care category until vosoritide approval in 2021. Vosoritide (a C-natriuretic peptide analogue) has commercial maturity in paediatric achondroplasia with growing real-world data on growth velocity and bone outcomes; follow-on CNP analogue programs and FGFR3-targeted programs are in late-stage trials. The question of whether disease-modifying therapy can also reduce the spinal and other complications of achondroplasia long-term is shaping the next-cycle commercial trajectory.