Description
This Phase 3 study randomised 121 children to vosoritide▼ or placebo for 52 weeks. Results showed a highly significant increase in annualised growth velocity and height Z scores – suggesting vosoritide is an effective treatment to increase growth in children with achondroplasia, although it is not yet known whether final adult height will be increased, or what the harms of long-term therapy might be.
EU-VOX-00243
References:
Savarirayan R, Tofts L, Irving M, Wilcox W, Bacino CA, Hoover-Fong J, Ullot Font R, Harmatz P, Rutsch F, Bober MB, Polgreen LE, Ginebreda I, Mohnike K, Charrow J, Hoernschemeyer D, Ozono K, Alanay Y, Arundel P, Kagami S, Yasui N, White KK, Saal HM, Leiva-Gea A, Luna-González F, Mochizuki H, Basel D, Porco DM, Jayaram K, Fisheleva E, Huntsman-Labed A, Day J Lancet 2020;396(10252):684–92