SAT-185 First Real-World Evidence Of Somapacitan Therapy Initiation In Children With GHD From GloBE-Reg

Abstract Disclosure: A. Al-Agha: None. A.K. Maniatis: Ascendis, Novo Nordisk, OPKO, Pfizer, Inc. D. Schnabel: Novo Nordisk. A. Pietropoli: Novo Nordisk. K. Belling: Novo Nordisk. S.F. Ahmed: Novo Nordisk, Pfizer, Inc., GenSci. P. Kotnik: Novo Nordisk. Background: Somapacitan is a once-weekly long-acting growth hormone (LAGH) therapy for children and adults with growth hormone deficiency (GHD). Real-world evidence (RWE) on LAGH therapies is crucial to evaluate long-term safety and efficacy but is currently limited. The Global Registry For Novel Therapies In Rare Bone & Endocrine Conditions (GloBE-Reg; globe-reg.net) is a new international registry for patients receiving growth hormone (GH) treatment. We used GloBE-Reg data to provide the first global RWE of children starting somapacitan treatment for GHD, to understand patient demographics, disease characteristics and dose patterns. Methods: As of Dec 2024, baseline data were available for 176 children starting somapacitan. Baseline characteristics were evaluated; results for each parameter were based on patients with data available. Puberty status was determined by Tanner Stage, and weight and height standard deviation scores (SDSs) were computed using WHO reference data; data up to 6 months after baseline were used. Somapacitan dose was calculated by dividing baseline dose by weight up to 6 months after baseline; a sensitivity analysis was performed using weight registered ±3 months from baseline. Results: Patients analyzed reside predominantly in the Middle East/Africa (75%), followed by Europe (15%) and North America (10%). Overall, 63% (111/176) of patients starting somapacitan treatment were male. Mean age (range) of patients (n=132) was 9.28 (2.33-15.39) years for GH-naïve (n=79) and 10.54 (3.94-16.42) years for non-naïve patients (n=53). Within ±6 months of baseline, 57% (66/115) of patients were pre-pubertal vs 43% (49/115) pubescent. Mean gestational age (range) of patients (n=135) was 38 (26-42) weeks. Mean (standard deviation, SD) birth weight SDS of patients (n=133) was -0.82 (1.32). The primary conditions reported were non-specific GHD (n=150; 85%), congenital idiopathic GHD (n=23; 13%) or organic GHD (n=3; 2%). Mean (SD) GH peak at baseline was 5.53 ng/mL (2.36; n=143). Mean (SD) height SDS was -1.66 (0.83) for GH-naïve (n=67) and -0.91 (1.07) for non-naïve patients (n=49). Mean (SD) IGF-1 SDS was -1.03 (1.32) for GH-naïve (n=76) and 0.62 (1.31) for non-naïve patients (n=23). Mean (SD) somapacitan dose at treatment start was 0.147 (0.016) and 0.154 (0.027) mg/kg/week for GH-naïve (n=61) and non-naïve patients (n=47), respectively. Conclusion: For the first time, RWE on children starting somapacitan for GHD are reported, offering valuable insights into their profiles. Consistent with expectations, most patients were male and around 10 years old, while baseline somapacitan dose was lower than the approved dose of 0.16 mg/kg/week. Notably, GH-naïve patients began treatment at or approaching puberty; studies suggest improved outcomes are associated with earlier treatment start. Presentation: Saturday, July 12, 2025