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Clinical Trial
. 2022;95(3):275-285.
doi: 10.1159/000524600. Epub 2022 Apr 13.

Efficacy and Safety of Once-Weekly Somatrogon Compared with Once-Daily Somatropin (Genotropin®) in Japanese Children with Pediatric Growth Hormone Deficiency: Results from a Randomized Phase 3 Study

Reiko Horikawa  1 , Toshiaki Tanaka  2 , Yukihiro Hasegawa  3 , Tohru Yorifuji  4 , David Ng  5 , Ron G Rosenfeld  6 , Yuko Hoshino  7 , Akifumi Okayama  7 , Daisuke Shima  8 , Roy Gomez  9 , Aleksandra Pastrak  10 , Orlando Castellanos  10
Affiliations
Clinical Trial

Efficacy and Safety of Once-Weekly Somatrogon Compared with Once-Daily Somatropin (Genotropin®) in Japanese Children with Pediatric Growth Hormone Deficiency: Results from a Randomized Phase 3 Study

Reiko Horikawa et al. Horm Res Paediatr. 2022.

Abstract

Introduction: Somatrogon is a long-acting recombinant human growth hormone being developed as a once-weekly treatment for children with growth hormone deficiency (GHD). The objective of this phase 3 study (NCT03874013) was to compare the efficacy and safety of once-weekly somatrogon with once-daily Genotropin in Japanese children with GHD.

Methods: In this open-label, randomized, active-controlled study, 44 prepubertal Japanese children with GHD (boys: 3 to <11 years; girls: 3 to <10 years) were randomized 1:1 to receive once-weekly somatrogon or once-daily Genotropin (0.025 mg/kg/day) for 12 months. Dose escalation for somatrogon-treated subjects occurred in the first 6 weeks (0.25, 0.48, and 0.66 mg/kg/week; 2 weeks each) with the remaining 46 weeks at a dose of 0.66 mg/kg/week. The study's primary endpoint was annualized height velocity (HV) at 12 months.

Results: Baseline characteristics were similar between treatment groups. Compared with Genotropin-treated subjects, somatrogon-treated subjects had higher least-squares mean HV at 12 months (9.65 cm/year vs. 7.87 cm/year). Once-weekly somatrogon was concluded as being comparable to once-daily Genotropin as the mean treatment difference (somatrogon-Genotropin) in HV was +1.79 cm/year (95% confidence interval, 0.97-2.61), which was greater than the preestablished margin (-1.8 cm/year). For both treatment groups, most adverse events were mild to moderate in severity and a similar proportion of subjects reported injection-site pain, although the somatrogon group reported more painful injections.

Conclusion: In prepubertal Japanese children with GHD, once-weekly somatrogon was comparable to once-daily Genotropin in terms of annualized (12-month) HV. Both treatments had similar safety and tolerability profiles.

Keywords: Genotropin; Growth hormone; Growth hormone deficiency; Long-acting growth hormone; Somatrogon.

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Conflict of interest statement

R. Horikawa: advisory boards of Novo Nordisk, Pfizer, Lumos Pharma, and OPKO; consulting fees from Novo Nordisk, Pfizer, Lumos Pharma, Sandoz, and Ascendis Pharma; recipient of grants from Novo Nordisk and Sandoz; speakers bureau for Novo Nor­disk, Pfizer, Eli Lilly & Company, and Sandoz. T. Tanaka: advisory board of OPKO and an Editorial Board Member of Hormone Research in Paediatrics. T. Yorifuji: advisory boards of Novo Nordisk and Pfizer; consulting fees from Novo Nordisk and Pfizer. R.G. Rosenfeld: advisory boards of Lumos, DNARx, and BioMarin; consulting fee from OPKO. Y. Hoshino, A. Okayama, D. Shima, and R. Gomez: employees and stockholders of Pfizer. A. Pastrak and O. Castellanos: employees and stockholders of OPKO. Y. Hasegawa and D. Ng: no conflicts of interest to declare.

Figures

Fig. 1
Fig. 1
Subject disposition. AE, adverse event.
Fig. 2
Fig. 2
Box plot of height velocity over time (full analysis set). Baseline defined as the last nonmissing measurement prior to the start of study drug. Missing values are not imputed.
Fig. 3
Fig. 3
Box plot of IGF-1 SDS over time (full analysis set). SDS, standard deviation score.
Fig. 4
Fig. 4
Median somatrogon serum concentration following once-weekly administration.
See this image and copyright information in PMC

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