Somapacitan Treatment in Children and Adolescents Born SGA: Two-Year Efficacy and Preference Over Daily GH

Abstract Context Children born small for gestational age (SGA) without spontaneous catch-up growth may require GH treatment; however, daily injections are burdensome. Somapacitan is a once-weekly long-acting GH. Objective To assess efficacy and safety of once-weekly somapacitan in children born SGA, including outcomes after switching from daily GH. Design REAL8 is a randomized, multinational, open-label, active-controlled phase 3 study with a 52-week main period and 104-week extension (NCT05330325). REAL9 (NCT05723835) is a multinational, open-label, uncontrolled phase 3 study. Setting Clinics across Europe, Asia, and the Americas. Participants REAL8 randomized 142 GH-treatment-naïve, prepubertal children born SGA; 141 received treatment and 139 completed 104 weeks. REAL9 enrolled 12 children/adolescents born SGA (GH-naïve or previously treated). Interventions In REAL8, participants were randomized (2:1:1) to somapacitan 0.24 mg/kg/week or daily GH (0.035 or 0.067 mg/kg/day) for 52 weeks; all received 0.24 mg/kg/week somapacitan in weeks 52–104. In REAL9, participants received 0.24 mg/kg/week somapacitan for 26 weeks. Main Outcome Measures Annualized height velocity (HV); secondary outcomes included height SD score (HSDS). Results In REAL8, mean HV during weeks 52–104 remained above baseline across groups (8.5–8.8 cm/year), including after switching from daily GH; HSDS increased through week 104, and IGF-I SDS were similar between groups. In REAL9, HV increased in GH-naïve participants and was maintained in previously treated participants. Somapacitan was well-tolerated; adverse events were mostly mild and injection-site reactions were infrequent. Most participants switching from daily GH preferred somapacitan. Conclusions In children born SGA, once-weekly somapacitan maintained efficacy through 2 years, with safety comparable to daily GH, and was preferred after switching.