TOPLINE:

Somapacitan, a long‑acting growth hormone (GH) derivative, given once weekly at 0.24 mg/kg/wk, was safe, well tolerated, and effective for up to 4 years in prepubertal children born small for gestational age with short stature, including those who switched from daily GH therapy.

METHODOLOGY:

• This study reported the long-term efficacy and safety of once-weekly somapacitan (0.24 mg/kg/wk) in prepubertal children born small for gestational age with short stature, including those who switched from daily GH therapy or lower doses of somapacitan.
• Initially, 62 treatment-naive children (64.5% boys) were randomly assigned to receive either somapacitan (0.16, 0.20, or 0.24 mg/kg/wk) or daily GH therapy (0.035 or 0.067 mg/kg/d) for 52 weeks.
• After 52 weeks, 60 children entered the extension phase, of whom 12 continued on somapacitan 0.24 mg/kg/wk and 48 switched to it from either daily GH therapy or lower doses of somapacitan; 55 children completed 208 weeks of treatment.
• GH activity was assessed by measuring insulin-like growth factor 1.
• Outcomes, assessed at prespecified visits through week 208, included height velocity; changes from baseline in height SD scores, height velocity SD scores, and the ratio of bone age to chronological age; and safety assessments.

TAKEAWAY:

• The mean height velocity at week 208 ranged from 6.6 to 7.7 cm/y across treatment groups, and the mean change from baseline in height velocity SD scores at week 208 ranged from 2.53 to 4.07.
• Height SD scores rose steadily in all groups, from mean baseline values between -2.92 and -3.15 to values within the normal range by week 208. For most children, the ratio of bone age to chronological age remained below 1.0.
• Insulin‑like growth factor 1 SD scores rose from baseline and were maintained through week 208. The overall safety profile of somapacitan 0.24 mg/kg/wk, including after switch, was similar to that of daily GH therapy.
• Among participants who switched from daily GH therapy, 87% preferred once‑weekly somapacitan; 80% of those respondents expected they would be more adherent to the weekly regimen.

IN PRACTICE:

"Four-year results from REAL5 show that somapacitan 0.24 mg/kg/week had a safety profile consistent with the well-established profile of daily GH treatment in SGA [small for gestational age]," the authors wrote.

SOURCE:

This study was led by Anders Juul, MD, PhD, DMSc, Copenhagen University Hospital-Rigshospitalet, Copenhagen, Denmark. It was published online on January 30, 2026, in the European Journal of Endocrinology.

LIMITATIONS:

Not all participants switched to the dose of 0.24 mg/kg/wk at the same time, which made direct comparisons of efficacy between groups difficult. The study had a small sample size.

DISCLOSURES:

This study was sponsored by Novo Nordisk A/S. One author reported being an employee of the sponsor and holding company stock. Several authors reported receiving honoraria, lecture fees, grant support, or consultancy payments from various pharmaceutical companies, including Novo Nordisk.

This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication.