hrp0095rfc7.2 | Growth and Syndromes | ESPE2022

Once-Weekly Somapacitan vs Daily GH in Children with GH Deficiency: The Randomized Phase 3 REAL 4 Trial

Miller Bradley , Blair Joanne , Højby Michael , Böttcher Volker , Juul Kildemoes Rasmus , Maniatis Aristides , Beck Bang Rikke , Mori Jun , Polak Michel , Stagi Stefano , Horikawa Reiko

Background: Growth hormone (GH) replacement therapy usually requires daily subcutaneous (s.c.) injections that can be burdensome for patients and their caregivers. Long-acting GH formulations aim to establish a less burdensome dosing regimen that is as safe and efficacious as daily GH to potentially improve adherence and clinical outcomes. Somapacitan, a long-acting reversible albumin-binding GH derivative, is in development for once-weekly s.c. administration...

hrp0095p1-305 | Growth and Syndromes | ESPE2022

Once-Weekly Somapacitan vs Daily Growth Hormone in Children Born Small for Gestational Age: 1-year Results from a Randomised Phase 2 Trial

Juul Anders , Backeljauw Philippe , Bakhtadze Bagci Ekaterine , Højby Michael , Kawai Masanobu , Juul Kildemoes Rasmus , Linglart Agnès , Zuckerman-Levin Nehama , Horikawa Reiko

Treatment of short stature in children born small for gestational age (SGA) requires daily growth hormone (GH) injections that can be burdensome for patients and caregivers. Once-weekly somapacitan is a long-acting GH in phase 3 development for replacement therapy in children with GH deficiency. We report the 52-week results of the first phase 2, multinational, randomised, open-label, controlled, dose-finding trial (NCT03878446) investigating efficacy and safety of somapacitan...

hrp0097fc14.5 | Late Breaking | ESPE2023

Predicting Average IGF-I Concentration for Once-Weekly Somapacitan in Children with Growth Hormone Deficiency

F. Backeljauw Philippe , Kamal Lyauk Yassine , C. Blair Joanne , S. Miller Bradley , Mori Jun , Højby Rasmussen Michael , Juul Kildemoes Rasmus

Growth hormone (GH) stimulates insulin like growth factor I (IGF I) release. IGF-I is the standard biomarker for monitoring GH effects during treatment and to achieve optimal long-term safety, and to a limited extent, monitor efficacy in children with GH deficiency (GHD). The IGF I profile during treatment with a long acting GH (LAGH), such as once weekly somapacitan (Novo Nordisk), differs from the daily GH profile by exhibiting larger peaks and troughs over the dosing interv...