The following is a summary of “Weekly Somapacitan in GH Deficiency: 4-Year Efficacy, Safety, and Treatment/Disease Burden Results From REAL 3,” published in the October 2023 issue of Endocrinology by Sävendahl, et al.

In the context of treating growth hormone deficiency (GHD) in children, the conventional approach involves daily growth hormone (GH) injections, which can pose challenges for both patients and their caregivers. Somapacitan, a GH derivative designed for weekly administration, has been under investigation for its efficacy and safety in the treatment of GHD.

For a study, researchers sought to evaluate the long-term (4 years) efficacy and safety of somapacitan, specifically examining the disease and treatment burden associated with its use. The research, an extension of a multicenter controlled phase 2 trial conducted at 29 sites across 11 countries, involved prepubertal, GH-naive children with GHD. Two groups of the fifty patients who completed the 4-year treatment were studied. The pooled group received varying doses of somapacitan (0.04, 0.08, 0.16 mg/kg/week) for the first year, followed by the highest dose (0.16 mg/kg/week) for the subsequent three years. The switched group, on the other hand, received daily GH 0.034 mg/kg/day for three years before transitioning to somapacitan 0.16 mg/kg/week for the final year. Key outcome measures included height velocity (HV), change in HV SD score (SDS) from baseline, change in height SDS from baseline, and assessments of disease and treatment burden for both patients and caregivers.

Results indicated that changes in HV and HV SDS from baseline were comparable in both groups, aligning with expectations. Observer-reported outcomes suggested a perceived reduction in treatment burden when transitioning from daily GH to somapacitan. The majority of parents/guardians (81.8%) expressed a strong preference for somapacitan over daily GH.

In conclusion, somapacitan demonstrated consistent efficacy and safety in patients continuing its use and those switching from daily GH. The transition to once-weekly injections with somapacitan alleviates the treatment burden compared to the daily injection regimen.

Source: academic.oup.com/jcem/article/108/10/2569/7095611