Somapacitan Treatment in Children and Adolescents Born SGA: Two-Year Efficacy and Preference Over Daily GH
Aristides Maniatis, Volker Böttcher, Michael Højby, Jan Frystyk, Xinran Cheng, Kenichi Kashimada, Anders Krogh Lemminger, Carlos Alberto Longui, Lawrence Silverman, Kamil Soltysik, Agnès LinglartAbstract
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.