EFFICACY, SAFETY, AND PATIENT-REPORTED OUTCOMES OF ONCE-WEEKLY SOMAPACITAN AND SOMATROGON IN CHILDREN WITH GROWTH HORMONE DEFICIENCY: A SYSTEMATIC REVIEW
Author(s)
Bander Balkhi, PharmD, PhD1, Faris E. Alotaibi, PharmD1, Nawaf Shalih Alqahtani, PharmD2.
1King Saud University, Riyadh, Saudi Arabia, 2National Guard Hospital, Riyadh, Saudi Arabia.
1King Saud University, Riyadh, Saudi Arabia, 2National Guard Hospital, Riyadh, Saudi Arabia.
OBJECTIVES: This review synthesises efficacy, safety, and patient-reported outcome (PRO) evidence on once-weekly somapacitan and somatrogon versus daily GH in paediatric GHD, with three objectives: (1) confirm non-inferior growth efficacy by height velocity (HV), height SDS, and IGF-1 SDS; (2) compare safety with attention to injection-site tolerability and immunogenicity; and (3) summarise PRO and HRQoL evidence across both programmes.
METHODS: A systematic search of PubMed/MEDLINE, Google Scholar and Scopus was conducted from inception to February 2026. Randomised controlled trials and their long-term extensions evaluating somapacitan or somatrogon versus daily GH in children with confirmed GHD were included.
RESULTS: Sixteen publications from eight unique clinical trials were included (somapacitan: n = 9 publications, 56%; somatrogon: n = 6 publications, 44%). Both agents demonstrated non-inferiority to daily GH for height velocity in Phase 3 trials: somapacitan (REAL4: HV 11.2 vs 11.7 cm/year; ETD −0.5, 95% CI −1.1 to 0.2; NI margin −1.8 cm/year) and somatrogon (Deal 2022: 10.10 vs 9.78 cm/year; mean difference +0.33, 95% CI −0.24 to 0.89). Overall adverse event profiles were broadly comparable. Injection-site pain was markedly more frequent with somatrogon (39-73% across Phase 3 trials) than with somapacitan (<2%); however, these rates were obtained using fundamentally different measurement methods, rendering direct comparison methodologically invalid. Anti-drug antibody rates were substantially higher with somatrogon (77-82%) than somapacitan (1.5-23%). Parental treatment burden was significantly reduced with somapacitan (GHD-PTB ETD −6.0, 95% CI −10.0 to −2.1, p = 0.003). No validated PRO instrument was used in pivotal somatrogon trials except one dedicated HRQoL study.
CONCLUSIONS: Both once-weekly GH formulations demonstrate non-inferior growth efficacy. The agents differ meaningfully in injection-site tolerability, immunogenicity, and the depth of PRO evidence. Somapacitan demonstrates significant and replicated reductions in parental treatment burden with strong caregiver preference for weekly dosing.
METHODS: A systematic search of PubMed/MEDLINE, Google Scholar and Scopus was conducted from inception to February 2026. Randomised controlled trials and their long-term extensions evaluating somapacitan or somatrogon versus daily GH in children with confirmed GHD were included.
RESULTS: Sixteen publications from eight unique clinical trials were included (somapacitan: n = 9 publications, 56%; somatrogon: n = 6 publications, 44%). Both agents demonstrated non-inferiority to daily GH for height velocity in Phase 3 trials: somapacitan (REAL4: HV 11.2 vs 11.7 cm/year; ETD −0.5, 95% CI −1.1 to 0.2; NI margin −1.8 cm/year) and somatrogon (Deal 2022: 10.10 vs 9.78 cm/year; mean difference +0.33, 95% CI −0.24 to 0.89). Overall adverse event profiles were broadly comparable. Injection-site pain was markedly more frequent with somatrogon (39-73% across Phase 3 trials) than with somapacitan (<2%); however, these rates were obtained using fundamentally different measurement methods, rendering direct comparison methodologically invalid. Anti-drug antibody rates were substantially higher with somatrogon (77-82%) than somapacitan (1.5-23%). Parental treatment burden was significantly reduced with somapacitan (GHD-PTB ETD −6.0, 95% CI −10.0 to −2.1, p = 0.003). No validated PRO instrument was used in pivotal somatrogon trials except one dedicated HRQoL study.
CONCLUSIONS: Both once-weekly GH formulations demonstrate non-inferior growth efficacy. The agents differ meaningfully in injection-site tolerability, immunogenicity, and the depth of PRO evidence. Somapacitan demonstrates significant and replicated reductions in parental treatment burden with strong caregiver preference for weekly dosing.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
CO7
Topic
Clinical Outcomes, Epidemiology & Public Health, Patient-Centered Research
Topic Subcategory
Clinician Reported Outcomes, Comparative Effectiveness or Efficacy
Disease
Diabetes/Endocrine/Metabolic Disorders (including obesity), No Additional Disease & Conditions/Specialized Treatment Areas, Pediatrics, Rare & Orphan Diseases