ESPE Abstracts

Growth hormone deficiency (GHD) in children results in reduced adult height. Children with GHD typically require daily subcutaneous (s.c.) injections of growth hormone (GH). Daily injections are burdensome for both patients and their caregivers and reduced adherence negatively impacts clinical outcomes. Somapacitan (Novo Nordisk) is a long-acting reversible albumin-binding human GH derivative in development for once-weekly s.c. administration in children with GHD, aiming to overcome the treatment burden of daily injections. REAL4 is a multi-national, randomized, open labelled phase 3 trial consisting of a 52-week main-phase followed by a single-group three-year extension (NCT03811535). Here we present a sub-analysis of Japanese GHD patients. Thirty patients from Japan were recruited into the study. During the main-phase, 11 received daily GH (0.034 mg/kg/day Norditropin®) and 19 patients received 0.16 mg/kg/week somapacitan. After 52-weeks of treatment, patients in the daily GH group switched to somapacitan (switch group) while patients receiving somapacitan continued treatment (soma/soma group). All patients participated for 104-weeks. Similar mean annualised height velocity (HV) was observed at week 52 between treatments (9.8 vs. 10.3 cm/year for daily GH and somapacitan, respectively). Similar values for the groups were also observed at week 104 (annualised HV of 7.9 vs. 7.4 cm/year for switch and soma/soma, respectively). Other height-related parameters supported sustained growth, including similar increased Height SDS change from baseline to week 104 (1.54 and 1.39 for switch and soma/soma, respectively). IGF-I SDS increased in both groups (observed mean IGF-I SDS at week 104 was 0.14 and -0.06 for switch and soma/soma groups, respectively) with mean IGF-I SDS being within -2 and +2 during the study. Somapacitan was well tolerated, with no safety or local tolerability issues identified. No neutralizing anti-somapacitan antibodies were detected. One mild injection site reaction was reported, with no reports of injection site pain. Patient preference questionnaires were responded to, in week 56, by all caregivers of patients in the switch group. Almost all (91%) preferred somapacitan, to some degree, with none preferring daily GH. The top reason given for this preference was the number of times needing to do injections. Of the 10 caregivers who preferred somapacitan, 6 believed they would be more adherent to somapactian over daily GH. In conclusion, once-weekly somapacitan showed sustained efficacy and was well-tolerated for 2 years in Japanese patients and a preference for somapacitan was reported in those who switched from daily GH.