ey0021.5-10 | Novel Treatments | ESPEYB21
Savarirayan Ravi
, Wilcox William R.
, Harmatz Paul
, Phillips John
, Polgreen Lynda E.
, Tofts Louise
, Ozono Keiichi
, et al.
In brief: This multinational, randomised, double-blind, placebo-controlled, Phase 2 study examined the safety and efficacy of vosoritide, a recombinant C-type natriuretic peptide (CNP) analogue, in 75 children with achondroplasia under age 5 years. Mean gain in height Z-score after 52-weeks was 025 (95% CI -002 to 053). The study was sponsored by BioMarin Pharmaceutical.Commentary: Achondroplasia is one of the most common constitutional...