Efficacy and Safety of Somapacitan vs Daily Growth Hormone in Children with Growth Hormone Deficiency: A Pooled Analysis of Randomised Trial Data

BACKGROUND: Several studies have individually suggested that somapacitan, a once-weekly growth hormone (GH) treatment, has similar efficacy and safety to once-daily GH in children with GH deficiency. OBJECTIVE: To assess the efficacy and safety of somapacitan vs daily GH after 52 and 156 weeks of treatment using pooled data from the REAL 3, REAL 4, and REAL 6 trials. METHODS: Participants receiving somapacitan 0.16 mg/kg/week or daily GH 0.034 mg/kg/day were included. Some participants switched from daily GH to somapacitan after 52 weeks of treatment, so data beyond week 52 were analysed separately. Outcomes including height velocity (HV) and adverse events (AEs) were analysed by treatment group. An additional analysis was conducted in relation to puberty onset and Tanner stage. RESULTS: At week 52, HV was similar in participants receiving somapacitan and those receiving daily GH. At week 156, mean (SD) HV (cm/year) was 7.5 (1.6) in participants who had always received somapacitan, 7.6 (2.0) in those who had always received daily GH, and 7.8 (1.4) in those who switched to somapacitan from daily GH. Most AEs were mild and unlikely to be treatment-related. HV remained stable for up to 130 weeks in pubertal children, and was similar across treatment groups and Tanner stages. CONCLUSIONS: In this pooled analysis of clinical trial data, somapacitan demonstrated similar efficacy and safety to daily GH for up to 156 weeks' treatment. A dose of 0.16 mg/kg/week of somapacitan was able to maintain consistent growth in children and adolescents who reached puberty.