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A Novel Y-Shaped Pegylated Recombinant Human Growth Hormone for Children With Growth Hormone Deficiency
Yan Liang1, Haiyan Wei2, Fan Yang3
1Department of Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan 430030, China.
Insights
Weekly Pegpesen® (pegylated recombinant human growth hormone) demonstrated non-inferiority to daily rhGH in treating pediatric growth hormone deficiency. This novel treatment offers a comparable safety profile and reduced dosage requirements for children.
Area of Science:
- Pediatric Endocrinology
- Pharmacology
- Clinical Trials
Background:
- Pegpesen® is a novel Y-shape pegylated recombinant human growth hormone (rhGH) for once-weekly treatment.
- Growth hormone deficiency (GHD) impacts children's growth and development.
Purpose of the Study:
- To evaluate the efficacy and safety of weekly Pegpesen® compared to daily rhGH in children with GHD.
- To establish Pegpesen® as a viable alternative therapeutic option.
Main Methods:
- A 52-week, multicenter, randomized, controlled phase 3 clinical trial.
- 391 pediatric participants with GHD were randomized 2:1 to weekly Pegpesen® or daily rhGH.
- Primary endpoint: growth velocity (GV) at 52 weeks; Secondary endpoints: changes in height standard deviation scores.
Main Results:
- Weekly Pegpesen® was non-inferior to daily rhGH in achieving GV (9.910 cm/year vs 10.037 cm/year).
- No significant differences were observed in height standard deviation scores (ΔHt SDS CA and ΔHt SDS BA) between groups.
- Comparable safety profiles and treatment adherence were reported for both Pegpesen® and daily rhGH.
Conclusions:
- Pegpesen® offers a non-inferior treatment option for pediatric GHD compared to daily rhGH.
- The study supports Pegpesen® as a safe and effective alternative with potentially lower dosage requirements.
- Pegpesen® presents a new therapeutic avenue for managing GHD in children.
Context:
Pegpesen is a novel Y-shape pegylated recombinant human growth hormone (rhGH) for once-weekly treatment of children with growth hormone deficiency (GHD).
Objective:
This work aimed to evaluate the efficacy and safety of Pegpesen in children with GHD vs daily rhGH.
Methods:
A multicenter, randomized, controlled phase 3 clinical trial was conducted at 23 centers in China with a duration of 52 weeks' treatment. There were 391 pediatric participants diagnosed with GHD. Participants were randomly assigned 2:1 to a weekly Pegpesen group (140 μg/kg/week) or a daily rhGH group (245 μg/kg/week) for 52 weeks. The primary end point was the growth velocity (GV) at 52 weeks, and the secondary end points mainly involved changes from baseline in height SD scores for chronological age and bone age (ΔHt SDS CA and ΔHt SDS BA).
Results:
At 52 weeks, the least squares mean (LS means) of GV was 9.910 cm/year in the Pegpesen group and 10.037 cm/year in the daily rhGH group. The LS means difference between groups was -0.127 (95% CI, -0.4868 to 0.2332), confirming that weekly Pegpesen is noninferior to daily rhGH. The LS means of ΔHt SDS CA, ΔHt SDS BA, were similar across both groups (all P > .05). Safety profiles and adherence were comparable.
Conclusion:
Pegpesen was noninferior to daily rhGH, with similar safety, lower dosage requirements, thus presenting a new therapeutic option for children with GHD.
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