FDA Approves Novo Nordisk's Sogroya for Three Additional Pediatric Growth Disorders
Key Insights
The FDA has approved Novo Nordisk's once-weekly growth hormone Sogroya (somapacitan-beco) for three new pediatric indications: idiopathic short stature (search), Noonan syndrome (search), and children born small for gestational age (search).
Sogroya demonstrated non-inferiority to daily growth hormone therapy in the REAL8 pivotal trial, achieving primary endpoints for mean annualized height velocity at 52 weeks across all three conditions.
The approval provides families with a once-weekly treatment alternative to daily injections, potentially addressing adherence challenges with 313 injection-free days per year.
The US Food and Drug Administration has approved three new pediatric indications for Novo Nordisk's once-weekly growth hormone therapy Sogroya (somapacitan-beco), expanding treatment options for children with growth disorders. The approvals cover children aged 2.5 years and older with idiopathic short stature (search) (ISS), short stature born small for gestational age (search) (SGA) with no catch-up growth by age 2, and growth failure associated with Noonan syndrome (search) (NS).
The approval makes Sogroya the first and only once-weekly, long-acting growth hormone available for these three conditions, offering an alternative to the daily injection regimen that has defined growth disorder treatment for more than 40 years. According to Novo Nordisk, this gives Sogroya the broadest range of approved indications among long-acting growth hormones.
Clinical Trial Results Support Efficacy
The FDA's decision was based on data from the REAL8 study, a multi-center, randomized, open-label, active-comparator, phase 3 basket study that demonstrated non-inferiority of once-weekly Sogroya compared to daily growth hormone treatment across all three indications.
In children with ISS, Sogroya demonstrated non-inferiority in mean annualized height velocity (AHV) compared with daily somatropin, achieving 10.2 cm/year versus 10.5 cm/year at week 52. For children born SGA with no catch-up growth by age 2, Sogroya showed a mean AHV of 11.0 cm/year compared to 9.4 cm/year and 11.1 cm/year for two different daily growth hormone doses. In children with growth failure associated with Noonan syndrome (search), Sogroya achieved a mean AHV of 10.4 cm/year versus 9.2 cm/year for daily somatropin.
The REAL8 study enrolled 307 pediatric patients across the three conditions. For SGA, 142 patients aged 2.6 to 10.7 years were randomized to receive Sogroya 0.24 mg/kg/week or one of two daily somatropin doses. The Noonan syndrome (search) arm included 77 patients aged 2 to 11.1 years, while the ISS group comprised 88 patients aged 2.8 to 10.8 years.
Safety Profile Consistent with Growth Hormone Class
In the REAL8 study, adverse reactions occurring in 10% or more of patients treated with Sogroya across all three indications included respiratory tract infection, nasopharyngitis, ear infection, and diarrhea. Additional reactions occurring in 10% or more of patients included headache in ISS and NS patients, cough, pyrexia, and vomiting in NS and SGA patients, and injection site reactions in ISS patients.
The safety profile aligns with known effects of growth hormone therapy, with no new safety signals identified in the pediatric populations studied.
Addressing Treatment Adherence Challenges
"Daily injections have defined the growth disorder treatment paradigm for more than 40 years," said Nicky Kelepouris, Rare Endocrine Disorders-US Medical Lead at Novo Nordisk. "Our scientific leadership and focus on advancing care in rare diseases led us to the development of Sogroya – a once‑weekly growth hormone therapy – which may help address the challenge of daily injections while offering patients and families a therapeutic option that delivers efficacy and safety."
Compliance with 365 daily injections per year for growth hormone treatment can be a common challenge for children and their caregivers. The once-weekly dosing schedule provides 313 injection-free days per year, potentially improving treatment adherence.
Dr. Aristides Maniatis, Founder of Rocky Mountain Pediatric Endocrinology and an investigator in the trial, noted that "families and healthcare professionals now have the option to consider a once-weekly growth hormone as treatment with 313 injection free days per year for their children 2.5 years and older with ISS, NS, and born SGA."
Expanding Treatment Landscape
Sogroya was previously approved in the US for adults with growth hormone deficiency (search) in August 2020 and for pediatric patients aged 2.5 years and older with growth failure due to inadequate secretion of endogenous growth hormone in April 2023. The new approvals significantly expand the eligible patient population for once-weekly growth hormone therapy.
Novo Nordisk has also submitted a supplemental application for Sogroya for approval in Turner syndrome (search), with a decision expected later this year. This would further broaden the treatment options available with the once-weekly formulation.
The approvals represent the culmination of decades of treatment innovation for growth disorders from Novo Nordisk and reflect the company's strategic focus on delivering evidence-based innovation for children living with growth disorders. The REAL8 study represents the first time a basket trial design of this type has been implemented in the growth disorder space, allowing for efficient evaluation across multiple related conditions.