Weekly somapacitan in GH deficiency : 4-year efficacy, safety, and treatment/disease burden results from REAL 3

Sävendahl, Lars (author), Battelino, Tadej (author), Højby Rasmussen, Michael (author), Brod, Meryl (author), Röhrich, Sebastian (author), Saenger, Paul (author), Horikawa, Reiko (author)

Context: Growth hormone deficiency (GHD) in children is currently treated with daily injections of GH, which can be burdensome for patients and their parents/guardians. Somapacitan is a GH derivative in development for once-weekly treatment of GHD. Objective: This work aimed to assess the efficacy and safety of somapacitan, and associated disease/treatment burden, after 4 years of treatment and 1 year after switching to somapacitan from daily GH. Methods: This long-term safety extension of a multicenter, controlled phase 2 trial (NCT02616562) took place at 29 sites in 11 countries. Patients were prepubertal, GH-naive children with GHD. Fifty patients completed 4 years of treatment. Patients in the pooled group received somapacitan (0.04, 0.08, 0.16 mg/kg/week) for 1 year, followed by the highest dose (0.16 mg/kg/week) for 3 years. Patients in the switched group received daily GH 0.034 mg/kg/day for 3 years, then somapacitan 0.16 mg/kg/week for 1 year. Main outcome measures were height velocity (HV), change from baseline in HV SD score (SDS), change from baseline in height SDS, disease burden, and treatment burden for patients and parents/guardians. Results: Changes from baseline in HV and HV SDS were similar and as expected in both groups. Observer-reported outcomes showed that patients and parents/guardians seem to have experienced a reduced treatment burden when switching from daily GH to somapacitan. Most parents/guardians (81.8%) strongly/very strongly preferred somapacitan over daily GH. Conclusions: Somapacitan showed similar efficacy and safety in patients who continued somapacitan treatment and those who switched from daily GH to somapacitan. Once-weekly injections may lead to a reduced treatment burden relative to once-daily injections. A plain-language summary of this work is available.

• Language: English
• Keywords: childhood growth hormone deficiency, growth hormone treatment, daily growth hormone, somapacitan, long-acting growth hormone
• Work type: Article
• Typology: 1.01 - Original Scientific Article
• Organization: MF - Faculty of Medicine
• Publication status: Published
• Publication version: Version of Record
• Year: 2023
• Number of pages: Str. 2569–2578
• Numbering: Vol. 108, iss. 10
• PID: 20.500.12556/RUL-152415
• UDC: 616-053.2
• ISSN on article: 0021-972X
• DOI: 10.1210/clinem/dgad183
• COBISS.SI-ID: 154830083
• Publication date in RUL: 24.11.2023

Record is a part of a journal

• Title: The journal of clinical endocrinology & metabolism
• Shortened title: J. clin. endocrinol. metab.
• Publisher: Oxford University Press, Endocrine Society
• ISSN: 0021-972X
• COBISS.SI-ID: 60951

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• License: CC BY-NC-ND 4.0, Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International
• Link: http://creativecommons.org/licenses/by-nc-nd/4.0/
• Description: The most restrictive Creative Commons license. This only allows people to download and share the work for no commercial gain and for no other purposes.

Projects

• Funder: Other - Other funder or multiple funders
• Funding programme: Novo Nordisk