Fig. 1: Line plot of mean annualized growth velocity shown in 6-month intervals starting in the baseline observation study and continuing through the randomized placebo-controlled study for 52 weeks and then into the extension study for a total of 104 weeks, displayed by treatment arm derived from observed data.

Numbers at each time point reflect mean annualized growth velocity in cm/year and the standard deviation. Orange and dotted blue lines represent annualized growth velocity for participants randomized to the placebo study treatment arm and the solid blue lines represent annualized growth velocity for participants in the vosoritide study treatment arm. After 52 weeks and completion of the phase 3 study, 119 children were enrolled into the extension study, where all participants received vosoritide at a dose of 15 μg/kg/day. Fifty-eight participants originally randomized to vosoritide continued vosoritide in the extension study. By week 104, n = 44 participants had standing height assessments available to determine six-month interval annualized growth velocity at the two-year analysis time point. Sixty-one participants crossed over from placebo to vosoritide in the extension study and n = 47 had standing height assessments available to determine the six-month interval annualized growth velocity at the 2-year analysis time point. The cause of the missing data is disruptions to study visits due to the COVID-19 pandemic, where many site visits were replaced by virtual visits.