US FDA grants Novo Nordisk three new pediatric indications for growth hormone Sogroya

The US FDA has approved three new pediatric indications for Sogroya (somapacitan-beco), Novo Nordisk’s once-weekly growth hormone, making it the first long-acting growth hormone cleared for use in children with idiopathic short stature, short stature associated with being born small for gestational age, and growth failure linked to Noonan syndrome. The decision gives the product the broadest indication range among long-acting growth hormones currently on the US market. For Novo Nordisk, the decision extends a franchise that began with Sogroya’s initial approval for adult growth hormone deficiency in 2020 and expanded to pediatric growth hormone deficiency in 2023. The company said it has also submitted a supplemental application for a fourth pediatric indication, Turner syndrome, with a decision expected later in 2026.

Approval details and context

The new indications cover children aged 2.5 years and older in three distinct populations: those with idiopathic short stature (ISS), those born small for gestational age (SGA) who have not achieved catch-up growth by age two, and those with growth failure associated with Noonan syndrome (NS). Sogroya is administered as a subcutaneous injection once weekly, available in 5 mg, 10 mg, or 15 mg pen presentations. Combined with the existing pediatric and adult growth hormone deficiency indications, these somapacitan pediatric indications now span five approved uses in the United States.

Two other once-weekly growth hormone products, Ascendis Pharma’s Skytrofa (lonapegsomatropin-tcgd) and Pfizer’s Ngenla (somatrogon-ghla), remain approved only for pediatric growth hormone deficiency and do not hold approvals for ISS, SGA, or Noonan syndrome.

Novo’s approvals rest on data from REAL8, a multicenter, randomized, open-label, active-comparator Phase III basket study conducted in treatment-naive, pre-pubertal children. The trial enrolled 307 patients across three sub-studies, each designed to test non-inferiority of once-weekly Sogroya (0.24 mg/kg/week) against daily somatropin on mean annualized height velocity (AHV) at 52 weeks.

In the ISS sub-study (n=88), Sogroya produced a mean AHV of 10.2 cm/year compared with 10.5 cm/year for daily somatropin at a dose of 0.05 mg/kg/day. In the SGA sub-study (n=142), Sogroya achieved a mean AHV of 11.0 cm/year, compared with 9.4 cm/year for a lower-dose daily somatropin arm (0.035 mg/kg/day) and 11.1 cm/year for a higher-dose arm (0.067 mg/kg/day). In the NS sub-study (n=77), mean AHV was 10.4 cm/year for Sogroya versus 9.2 cm/year for daily somatropin at 0.05 mg/kg/day. Non-inferiority was met across all three sub-studies.

Daily recombinant human growth hormone injections have served as the standard treatment for pediatric growth disorders for more than four decades. Multiple somatropin products, including Novo Nordisk’s own Norditropin, Pfizer’s Genotropin, and Eli Lilly’s Humatrope, hold approvals across various combinations of ISS, SGA, Turner syndrome, and Noonan syndrome. The therapeutic challenge in these populations has not been a lack of available molecules but rather the burden of daily injections, which published literature has linked to adherence difficulties in pediatric patients and their caregivers.

The arrival of once-weekly formulations has begun to shift this landscape, though unevenly. Skytrofa received US FDA approval for pediatric growth hormone deficiency in 2021, and Ngenla followed in 2023, but neither has secured approval for ISS, SGA, or Noonan syndrome. Ascendis Pharma is currently recruiting for a Phase III ISS program for lonapegsomatropin, and an investigator-sponsored phase trial comparing somatrogon to daily somatropin in ISS and SGA is also recruiting. For now, Sogroya stands alone as a once-weekly option approved for long-acting growth hormone treatment in children with these three conditions.