Somapacitan effective and safe for children with Growth Hormone deficiency, finds a Study

Somapacitan was found to be effective and safe compared to growth hormone in growth hormone deficient children as per a recent study that was published in the Journal of Clinical Endocrinology and Metabolism. 

Growth deficiency can occur at different ages, depending on various mechanisms like hormonal deficiency or genetic conditions, or idiopathic. Somapacitan is a reversible albumin-binding GH derivative. It is given at a once-weekly dosage. Recently researchers evaluated the efficacy and safety of the drug Somapacitan in children with GH deficiency (GHD) in comparison with growth hormone. 

A randomized, multi-national, open-labeled, active-controlled parallel-group phase 3 trial called the REAL4 trial was done at eighty-six sites across 20 countries. REAL4 comprised a 52-week main trial and a three-year extension. 200 treatment-naïve participants were randomized 2:1 to somapacitan at 0.16 mg/kg/week or daily GH at 0.034 mg/kg/day, administered subcutaneously. Norditropin® was the GH that was given to the participants. The primary endpoint was annualized height velocity (HV; cm/year) at week 52. Additional assessments included HV standard deviation score (SDS), height SDS, bone age, IGF-I SDS, patient-reported outcomes, and safety measures. 

Key findings: 

• Estimated mean HV at week 52 was 11.2 and 11.7 cm/year for somapacitan and daily GH, respectively.
• Somapacitan was Non-inferior to GH.
• Changes in HVSDS, height SDS, bone age, and IGF-I SDS from baseline to week 52 were similar between treatment groups.
• At week 52, mean IGF-I SDS values were similar between treatment groups and within the normal range (-2 to +2).
• Safety of somapacitan was consistent with the well-known daily GH profile.
• Low proportions of injection-site reactions were reported for somapacitan (5.3%) and daily GH (5.9%).
• Both treatments similarly reduced disease burden from baseline to week 52, while a greater treatment burden reduction was observed for somapacitan.  

Thus, the researchers concluded that somapacitan showed similar efficacy to GH and was also safe over 52 weeks of treatment and demonstrated mean IGF-I SDS levels in treatment-naïve children with GHD. 

For further reading, click here: https://doi.org/10.1210/clinem/dgac513

Miller BS, Blair JC, Rasmussen MH, et al. Weekly Somapacitan is Effective and Well Tolerated in Children with GH Deficiency: The Randomized Phase 3 REAL4 Trial [published online ahead of print, 2022 Sep 5]. J Clin Endocrinol Metab. 2022;dgac513.