Ann Pediatr Endocrinol Metab.  2025 Feb;30(1):11-16. 10.6065/apem.2448258.129.

Somatrogon in pediatric growth hormone deficiency: a comprehensive review of clinical trials and real-world considerations

Affiliations
  • 1Rocky Mountain Pediatric Endocrinology, Centennial, CO, USA
  • 2Senior Medical Director, Pfizer Ltd., NY, USA
  • 3New York University Grossman School of Medicine, New York, NY, USA
  • 4Medical Affairs Director, Pfizer Ltd., Tadworth, UK
  • 5Senior Medical Manager, Pfizer Ltd., Seoul, Korea
  • 6Senior Medical Affairs Scientist, Pfizer Ltd., Seoul, Korea

Abstract

Growth hormone (GH) is crucial for childhood growth and body composition. In pediatric GH deficiency (pGHD), the pituitary gland fails to produce sufficient GH, which affects linear growth in childhood. pGHD is conventionally treated with daily recombinant human GH (rhGH); however, because GH therapy lasts throughout childhood, adherence to daily rhGH treatment can be low, resulting in suboptimal effectiveness. Somatrogon is a long-acting GH analog designed to address the challenges associated with daily GH therapy for pGHD. Somatrogon administered once per week is a potential alternative to daily GH therapy. The use of somatrogon is supported by phase II and III clinical trials demonstrating that once-weekly injections are noninferior to once-daily somatropin injections in terms of efficacy, safety, and tolerability and have the advantage of reduced treatment burden. This review summarizes the clinical trials of somatrogon and discusses the therapeutic profile and effects of treating pGHD with reduced injection frequency.

Keyword

Adherence; Pediatric growth hormone deficiency; Long-acting growth hormone; Somatrogon; Somatropin

Cited by  1 articles

Commentary on "Somatrogon in pediatric growth hormone deficiency: a comprehensive review of clinical trials and real-world considerations"
Yoo-Mi Kim
Ann Pediatr Endocrinol Metab. 2025;30(1):1-2.    doi: 10.6065/apem.2524129edi01.


Reference

References

1. Zelinska N, Iotova V, Skorodok J, Malievsky O, Peterkova V, Samsonova L, et al. Long-acting C-terminal peptide–modified hGH (MOD-4023): results of a Safety and Dose-Finding Study in GHD children. J Clin Endocrinol Metab. 2017; 102:1578–87.
Article
2. Deal CL, Steelman J, Vlachopapadopoulou E, Stawerska R, Silverman LA, Phillip M, et al. Efficacy and safety of weekly somatrogon vs daily somatropin in children with growth hormone deficiency: a phase 3 study. J Clin Endocrinol Metab. 2022; 107:e2717. –28.
Article
3. Stabler B. Impact of growth hormone (GH) therapy on quality of life along the lifespan of GH-treated patients. Horm Res. 2001; 56 Suppl 1:55–8.
Article
4. Horikawa R, Tanaka T, Hasegawa Y, Yorifuji T, Ng D, Rosenfeld RG, et al. Efficacy and safety of once-weekly somatrogon compared with once-daily somatropin (Genotropin(R)) in Japanese children with pediatric growth hormone deficiency: results from a randomized phase 3 study. Horm Res Paediatr. 2022; 95:275–85.
Article
5. Geisler A, Lass N, Reinsch N, Uysal Y, Singer V, Ravens-Sieberer U, et al. Quality of life in children and adolescents with growth hormone deficiency: association with growth hormone treatment. Horm Res Paediatr. 2012; 78:94–9.
Article
6. Saenger PH, Mejia-Corletto J. Long-acting growth hormone: an update. Endocr Dev. 2016; 30:79–97.
Article
7. Maniatis AK, Carakushansky M, Galcheva S, Prakasam G, Fox LA, Dankovcikova A, et al. Treatment burden of weekly somatrogon vs daily somatropin in children with growth hormone deficiency: a randomized study. J Endocr Soc. 2022; 6:bvac117.
Article
8. Cutfield WS, Derraik JGB, Gunn AJ, Reid K, Delany T, Robinson E, et al. Non-compliance with growth hormone treatment in children is common and impairs linear growth. PLoS One. 2011; 6:e16223.
Article
9. Christiansen JS, Backeljauw PF, Bidlingmaier M, Biller BM, Boguszewski MC, Casanueva FF, et al. Growth Hormone Research Society perspective on the development of long-acting growth hormone preparations. Eur J Endocrinol. 2016; 174:C1–8.
Article
10. Velazquez EP, Miller BS, Yuen KCJ. Somatrogon injection for the treatment of pediatric growth hormone deficiency with comparison to other LAGH products. Expert Review of Endocrinol Metab. 2024; 19:1–10.
Article
11. Loftus J, Quitmann J, Valluri SR. Health-related quality of life in pre-pubertal children with pediatric growth hormone deficiency: 12-month results from a phase 3 clinical trial of once-weekly somatrogon versus once-daily somatropin. Curr Med Res Opin. 2024; 40:175–84.
Article
12. Cohen P, Rogol AD, Howard CP, Bright GM, Kappelgaard AM, Rosenfeld RG, et al. Insulin growth factor-based dosing of growth hormone therapy in children: a randomized, controlled study. J Clin Endocrinol Metab. 2007; 92:2480–6.
Article
13. Zadik Z, Zelinska N, Iotova V, Skorodok Y, Malievsky O, Mauras N, et al. An open-label extension of a phase 2 dose-finding study of once-weekly somatrogon vs. once-daily Genotropin in children with short stature due to growth hormone deficiency: results following 5 years of treatment. J Pediatr Endocrinol Metab. 2023; 36:261–9.
Article
14. Fisher DM, Rosenfeld RG, Jaron-Mendelson M, Amitzi L, Koren R, Hart G. Pharmacokinetic and pharmacodynamic modeling of MOD-4023, a long-acting human growth hormone, in growth hormone deficiency children. Horm Res Paediatr. 2017; 87:324–32.
Article
15. Peter F, Bidlingmaier M, Savoy C, Ji HJ, Saenger PH. Three-year efficacy and safety of LB03002, a once-weekly sustained-release growth hormone (GH) preparation, in prepubertal children with GH deficiency (GHD). J Clin Endocrinol Metab. 2012; 97:400–7.
Article
16. Cohen P, Germak J, Rogol AD, Weng W, Kappelgaard AM, Rosenfeld RG. Variable degree of growth hormone (GH) and insulin-like growth factor (IGF) sensitivity in children with idiopathic short stature compared with GH-deficient patients: evidence from an IGF-based dosing study of short children. J Clin Endocrinol Metab. 2010; 95:2089–98.
Article
17. Hershkovitz O, Bar-Ilan A, Guy R, Felikman Y, Moschcovich L, Hwa V, et al. In vitro and in vivo characterization of MOD-4023, a long-acting carboxy-terminal peptide (CTP)-modified human growth hormone. Mol Pharm. 2016; 13:631–9.
18. Brod M, Alolga SL, Beck JF, Wilkinson L, Hojbjerre L, Rasmussen MH. Understanding burden of illness for child growth hormone deficiency. Qual Life Res. 2017; 26:1673–86.
Article
19. Maghnie M, Orso M, Polistena B, Cappa M, Pozzobon G, d'Angela D, et al. Quality of life in children and adolescents with growth hormone deficiency and their caregivers: an Italian survey. J Endocrinol Invest. 2023; 46:2513–23.
Article
Full Text Links
  • APEM
Actions
Cited
CITED
export Copy
Close
Share
  • Twitter
  • Facebook
Similar articles
Copyright © 2025 by Korean Association of Medical Journal Editors. All rights reserved.     E-mail: koreamed@kamje.or.kr