Registrational clinical program
Summaries below describe the registrational program for Pegpesen® (inpegsomatropin). Read them alongside the peer-reviewed Phase III manuscript on the Publications page.
The registrational program under NCT04513171 began with a 12-week Phase II PK/PD and height-velocity assessment at three once-weekly doses vs daily Norditropin—summarized on the Phase II study page.
For modeled dose up-titration and weight-banded scenarios built from the same trial data (not the same as the registrational primary analysis), see the dedicated PK/PD modeling topic.
Phase III confirmatory study
A randomized, open-label, active-controlled, multicenter trial evaluated safety and efficacy in 391 treatment-naïve pediatric participants with GHD who were >3 years, prepubertal (boys ≤11 years, girls ≤10 years), and had not received prior GH promoting therapy. Randomization 2:1 to:
- Pegpesen® 0.14 mg/kg/week, once weekly (N=261)
- Norditropin® 0.035 mg/kg/day SC once daily for 7 consecutive days (total weekly exposure 0.245 mg/kg/week; N=130)
Treatment duration 52 weeks. Primary efficacy endpoint: annualized growth velocity (GV) at Week 52.
Baseline characteristics (label)
Age 3.09–11.00 years (mean 6.796 years); 66.2% male, 33.8% female. Mean baseline GV (SD) 3.51 (1.373) cm/year.
Primary analysis — growth velocity
After 52 weeks, least squares means (LSMEANS) of GV were 9.910 cm/year (Pegpesen®) and 10.037 cm/year (Norditropin®). The least squares mean difference (Pegpesen minus Norditropin) was −0.127 cm/year (95% CI: −0.4868, 0.2332), demonstrating non-inferiority of Pegpesen® to Norditropin® (lower CI bound −0.4868 exceeded the pre-specified non-inferiority margin of −1.8 cm/year).
Height SDS
After 52 weeks, Ht SDS was −1.683 vs −1.654; change from baseline LSMEANS 0.873 vs 0.902 (difference −0.029; 95% CI −0.0955, 0.0375). Pegpesen® increased GV and improved Ht SDS with comparable Ht SDS changes vs Norditropin®.
Table: efficacy at Week 52 (full analysis set)
| Endpoint | Pegpesen® LSMEAN (N=261) | Norditropin® LSMEAN (N=130) | Difference (95% CI) |
|---|---|---|---|
| Growth velocity (cm/year) | 9.910 | 10.037 | −0.127 (−0.4868, 0.2332) |
| Ht SDS | −1.683 | −1.654 | −0.029 (−0.0955, 0.0375) |
| Change in Ht SDS from baseline | 0.873 | 0.902 | −0.029 (−0.0955, 0.0375) |
ANCOVA model (per label): treatment group, baseline GV or baseline Ht SDS, sex, baseline GH Cmax, and study center as covariates.
Phase II program (label summary)
Multicenter, randomized, open-label, active-controlled; total duration 2 years. Year 1: 43 patients aged ≥3 years randomized 1:1:1:1 to Pegpesen® 0.1, 0.12, or 0.14 mg/kg/week (weekly) vs Norditropin® 0.245 mg/kg/week (daily dosing schedule per protocol), each 52 weeks + 5-week follow-up. Year 2: all received Pegpesen® 0.14 mg/kg/week for 52 weeks + follow-up. Year-1 safety profile similar to Phase III; year-2 SOC breakdown in label (see adverse events page).