Novo Nordisk's Once-Weekly Sogroya Shows Efficacy in Children with Growth Disorders in Phase 3 Trial
核心洞察
The REAL8 phase 3 trial demonstrated that once-weekly Sogroya (somapacitan) was non-inferior to daily growth hormone in children with growth disorders, with superior results in Noonan syndrome (搜索) patients.
Sogroya's weekly administration could significantly improve treatment adherence, addressing a critical challenge where missed daily injections can result in a 6.1 cm height difference over three years.
Based on positive REAL8 and REAL9 trial data, Novo Nordisk has submitted regulatory applications for Sogroya in three pediatric indications to both EU and US authorities in April 2025.
Novo Nordisk's phase 3 REAL8 trial has demonstrated that once-weekly Sogroya® (somapacitan) is as effective as daily growth hormone therapy in treating children with various growth disorders, potentially offering a more convenient treatment option that could improve adherence and outcomes.
The 52-week study showed that Sogroya was non-inferior to once-daily Norditropin® (somatropin) in improving yearly growth rate in pre-pubertal children born small for gestational age (搜索) (SGA), those with Noonan syndrome (搜索) (NS), and children with idiopathic short stature (搜索) (ISS). Notably, Sogroya demonstrated superiority in children with Noonan syndrome and when compared to lower doses of daily growth hormone in children born SGA.
"These findings represent a significant advancement in pediatric growth hormone therapy," said Professor Agnès Linglart from Bicêtre Paris-Saclay University and Hospital, one of the lead investigators on REAL8. "Children with growth failure face numerous health challenges beyond height, including metabolic disruptions and developmental difficulties that can seriously affect their wellbeing and quality of life."
Key Efficacy Results
The REAL8 trial achieved its primary endpoints across all three indications presented:
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In children born SGA, Sogroya demonstrated superior estimated mean height velocity (HV) when compared with a lower dose of somatropin (11.0 vs 9.4 cm/year), and non-inferior estimated mean HV when compared with a higher dose (11.0 vs 11.1 cm/year).
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In children with Noonan syndrome (搜索), Sogroya showed superior estimated mean HV compared with somatropin (10.4 vs 9.2 cm/year).
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In children with idiopathic short stature (搜索), Sogroya demonstrated non-inferior estimated mean HV compared with daily somatropin (10.5 vs 10.5 cm/year).
The safety profile of Sogroya was comparable to that of daily growth hormone, with no new safety or tolerability issues identified. Insulin-like growth factor 1 (IGF-1) response in patients treated with once-weekly Sogroya was similar to those treated with daily growth hormone across all indications.
Addressing Treatment Adherence Challenges
A critical issue in growth hormone therapy is treatment adherence. Daily injections can be burdensome for children and caregivers, leading to missed doses due to discomfort, pain at injection sites, or disruption to daily routines. Research has shown that non-adherence can result in a 6.1 cm difference in height over three years compared to adherent patients.
Martin Lange, Executive Vice President for Development at Novo Nordisk, emphasized this challenge: "Imagine if a child misses only one day of treatment each week, amounting to 52 missed days per year. Over a seven-year treatment window, this results in one year of missed treatment and can have a significant knock-on impact on their health."
Innovative Trial Design
The REAL8 study employed an innovative basket trial design, investigating Sogroya in four different but related indications (SGA, Turner syndrome (搜索), Noonan syndrome (搜索), and ISS) under one trial protocol. This approach allowed for a more efficient clinical development process by speeding up recruitment and consolidating resources. Results from the Turner syndrome sub-study are expected later this year.
In the trial, pre-pubertal children with NS, Turner syndrome (搜索), or ISS were randomized to receive either once-weekly Sogroya 0.24 mg/kg/week or once-daily somatropin 0.050 mg/kg/day. Children born SGA were randomized to receive either Sogroya 0.24 mg/kg/week, or low dose of somatropin 0.035 mg/kg/day, or high dose of somatropin 0.067 mg/kg/day.
Technology Behind Sogroya
Sogroya is a human growth hormone analogue that uses albumin-binding prolongation technology. This innovative approach allows the growth hormone to attach to albumin, a protein in the blood, delaying its removal from the body and enabling once-weekly dosing. This technology has been used for over 20 years in Novo Nordisk's diabetes treatments.
Regulatory Status
Based on the positive data from REAL8 and the ongoing REAL9 study, Novo Nordisk submitted applications for the three indications (SGA, NS, and ISS) for regulatory review in both the European Union and United States in April 2025. Sogroya is currently approved for once-weekly treatment of children and adults who do not produce enough growth hormone.
Future Implications
If approved for these additional indications, Sogroya could significantly reduce the treatment burden for children with growth disorders and their families. The once-weekly administration schedule may improve adherence rates, potentially leading to better growth outcomes and quality of life for affected children.
The data from REAL8 were presented at the first Joint Congress of the European Society for Paediatric Endocrinology (ESPE) and European Society of Endocrinology (ESE) in Copenhagen, Denmark, in May 2025.