Somapacitan demonstrates sustained efficacy, patient preference in treating GHD in children
After 2 years of treatment and a switch from daily growth hormone (GH), somapacitan exhibited sustained efficacy and was preferred by the majority of patients and caregivers, according to findings of a Phase 3 trial.
The study, a randomized, multi-national, open-label, controlled parallel group trial, spanned 85 sites across 20 countries and included 200 treatment-naïve pre-pubertal patients initially enrolled and randomized, with 194 completing the two-year period.
During the first year, patients were randomized in a 2:1 ratio to receive either somapacitan (0.16 mg/kg/week) or daily GH (0.034 mg/kg/day). After the initial year, all patients were transitioned to somapacitan 0.16 mg/kg/week. The primary outcome measure was height velocity (HV) at week 104, with additional assessments including HV standard deviation score (SDS), height SDS, insulin-like growth factor-I (IGF-I) SDS, and observer-reported outcomes.
The results revealed that HV was sustained in both groups between weeks 52 and 104. At week 104, the mean HV between weeks 52 and 104 was 8.4 (1.5) cm/year after continuous somapacitan treatment and 8.7 (1.8) cm/year following the switch from daily GH. Secondary height-related endpoints also supported the sustained growth observed with somapacitan. Additionally, the mean IGF-I SDS during the second year was similar between the 2 groups and within the normal range of -2 to +2.
Miller BS, Blair JC, Rasmussen MH, et al. Effective GH Replacement with Somapacitan in Children with GHD: REAL4 2-year Results and after Switch from Daily GH. J Clin Endocrinol Metab. 2023;dgad394. doi: 10.1210/clinem/dgad394. Epub ahead of print. PMID: 37406251.