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Long-acting once-weekly growth hormone treatment has moved closer to expanded use in Europe for two new pediatric indications, potentially reducing injection frequency for children with specific growth disorders. 

The European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use has adopted a positive opinion recommending the expansion of approved indications for Sogroya (somapacitan, Novo Nordisk) for growth disturbances in children born small for gestational age (SGA) and for children with Noonan syndrome, in addition to its existing pediatric indication for growth hormone deficiency.

Who Qualifies for Treatment 

For SGA patients, the indication covers children with current height standard deviation score (SDS) below -2.5 and parental adjusted height SDS below -1, who were born with birth weight or length below -2 standard deviations and failed to show catch-up growth — defined as height velocity SDS below 0 during the last year — by 4 years of age or later.

Noonan syndrome affects approximately 1 in 1000-2500 live births and commonly causes short stature in up to 70% of affected children.

How the Weekly Hormone Injection Works 

Sogroya contains somapacitan as the active ingredient and is a long-acting growth hormone derivative. By extending growth hormone exposure, somapacitan allows once-weekly administration rather than daily injections. This reduces injection frequency from 365 daily injections to 52 weekly administrations per year.

The medication is administered subcutaneously using a prefilled pen, with injection sites rotated weekly among the abdomen, thighs, buttocks, or upper arms. Patients or caregivers can self-administer after appropriate training.

Treatment requires initiation and monitoring by qualified physicians experienced in managing growth hormone disorders, such as endocrinologists.

Growth Velocity Outcomes 

The broadened indications stem from clinical evidence demonstrating somapacitan’s efficacy in non-growth hormone deficient children. The phase 3 REAL8 trial evaluated once weekly somapacitan in children with growth disorders, including children born SGA and those with Noonan syndrome.

For children born SGA, the 52-week REAL8 substudy enrolled 142 prepubertal, treatment-naive children. Results showed somapacitan 0.24 mg/kg/wk achieved superior height velocity compared with daily growth hormone 0.035 mg/kg/d (11.0 vs 9.4 cm/y) and demonstrated noninferiority to daily growth hormone 0.067 mg/kg/d (11.0 vs 11.1 cm/y). 

The Noonan syndrome substudy included 77 treatment-naive, prepubertal children randomized to receive somapacitan 0.24 mg/kg/wk or daily growth hormone 0.050 mg/kg/d. Results demonstrated noninferiority and superiority for annualized height velocity at week 52, with estimated mean annualized height velocity of 10.4 cm/y vs 9.2 cm/y for daily therapy. 

Safety Profile and Contraindications 

Safety data from clinical trials showed somapacitan was well tolerated with adverse event profiles similar to daily growth hormone treatments. 

The most common side effect was headache, affecting more than 1 in 10 patients. Other common side effects include hypothyroidism, reactions at the injection site, peripheral edema, joint pain, hyperglycemia, tiredness, and adrenocortical insufficiency. In children and adolescents, another common side effect is pain in the arms and legs.

The treatment is contraindicated in patients with active tumors and must not be used to promote growth in children whose bones have finished growing.

This medicine was designated an orphan medicine and remains under additional monitoring.

Detailed recommendations for product use will be described in the updated summary of product characteristics, which will be published on the EMA website after the European Commission grants the marketing authorization change.