Significantly greater annualized growth velocity (AGV) at Week 52 compared with placebo1
Increases in AGV at Week 52 were greater with YUVIWEL vs placebo, regardless of age1:
- <5 years of age: +1.0 cm/year greater mean AGV
- ≥5 years of age: +1.8 cm/year greater mean AGV
Note: Data presented are least-square (LS) means unless otherwise noted.1
*Treatment differences between YUVIWEL and placebo were estimated from an analysis of covariance (ANCOVA) model that included treatment, strata (defined by sex and age), baseline age, and baseline achondroplasia-specific height Z-score as covariates.1
CI=confidence interval.
Long-term treatment effect1:
AGV was maintained throughout 2 years of treatment with YUVIWEL 0.1 mg/kg once weekly in a single-arm OLE of Trial 2
Trial 2 was a 52-week, randomized, double-blind, placebo-controlled, dose-finding trial that included 57 participants with achondroplasia (aged 2 to 10 years). At Week 52, all participants transitioned to an OLE period and received YUVIWEL 0.1 mg/kg/week for 104 weeks.1
YUVIWEL improved height Z-scores vs placebo at Week 521
Achondroplasia height Z-score: Change from baseline at Week 521,2*
CDC height Z-score: Change from baseline at Week 521,2‡§
Note: Data presented are least-square (LS) means unless otherwise noted.1
*Calculated using the reference data from untreated patients with achondroplasia (CLARITY Natural History Study).1
†Treatment differences between YUVIWEL and placebo were estimated from an analysis of covariance (ANCOVA) model that included treatment, strata (defined by sex and age), baseline age, and baseline achondroplasia-specific height Z-score as covariates.1
‡Calculated using the Centers for Disease Control and Prevention (CDC) reference data from the general population.1
§The endpoint was analyzed outside of the pre-specified multiple testing strategy.1
SE=standard error.
