Ngenla (somatrogon-ghla) / OPKO Health, Pfizer - LARVOL DELTA

Comparing the efficacy and safety of weekly somatrogon with daily somatropin in Asian children living with growth hormone deficiency: a plain language summary of publication. (PubMed, Ther Adv Endocrinol Metab) - "The results of this study may differ from those of other studies. Health professionals should make treatment decisions based on all available evidence and not on the results of a single study."

Journal • Review • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Pediatric) • Pediatrics • IGF1

Reversible Injection-Site Lipoatrophy Induced by Long-Acting Growth Hormone (Somatrogon) in Pediatric Growth Hormone Deficiency: A Case Series. (PubMed, J Clin Res Pediatr Endocrinol) - "Treatment was continued while injection sites were rotated to the thighs and abdomen. Complete resolution of lipoatrophy was achieved within three months, with no recurrence during follow-up."

Journal • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Pediatric) • Lipodystrophy • Pain • Pediatrics

Korean Post-marketing Surveillance Ngenla® Pre-filled Pen Injection for the Treatment of Pediatric Patients With Growth Disturbance Due to Insufficient Secretion of Growth Hormone (clinicaltrials.gov) - P=N/A | N=565 | Active, not recruiting | Sponsor: Pfizer | Trial completion date: Jan 2028 ➔ Sep 2026 | Trial primary completion date: Jan 2028 ➔ Sep 2026

Trial completion date • Trial primary completion date • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Pediatric) • Pediatrics

INTRODUCTION OF SOMATROGON IN PEDIATRIC GROWTH HORMONE DEFICIENCY: REAL-WORLD INSIGHTS FROM A NATIONAL SURVEY OF PEDIATRIC ENDOCRINOLOGISTS. (PubMed, Endocr Pract) - "This survey revealed heterogenous LAGH prescribing behaviors among pediatric endocrinologists during the introductory period of its availability. The findings call for continued search for real-world evidence and refined clinical guidelines to promote effective and safe integration of this promising therapy into clinical practice."

Journal • Real-world evidence • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Pediatric) • Lipodystrophy • Pediatrics

MISSION: Comparison of Weekly Somatrogon to Daily Genotropin in Children Born Small for Gestational Age or With Idiopathic Short Stature. (clinicaltrials.gov) - P3 | N=254 | Not yet recruiting | Sponsor: Rabin Medical Center

New P3 trial • Idiopathic Short Stature • Pediatrics • Small for Gestational Age • IGF1

Efficacy and safety of once-weekly somatrogon following up to 4 years of treatment in Japanese children with growth hormone deficiency: results from an open-label extension of a phase 3 study. (PubMed, Endocr J) - P3 | "Most TEAEs were mild or moderate in severity and no patients discontinued from the OLE or required dose reductions due to TEAEs. Up to 4 years of treatment with once-weekly somatrogon resulted in improved growth response and was well tolerated in Japanese patients with pediatric GHD, including patients who switched to somatrogon from once-daily somatropin.Clinialtrials.gov:NCT03874013."

Journal • P3 data • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Pediatric) • Pediatrics

Real-World Efficacy of Weekly Somatrogon on Growth and Bone Health in Pediatric Growth Hormone Deficiency: A 12-Month Retrospective Cohort Study. (PubMed, J Clin Res Pediatr Endocrinol) - "Weekly Somatrogon significantly improved linear growth, IGF-1 levels, and cortical bone health without advancing bone age in children with GHD. These findings support the efficacy of long-acting GH therapy and its potential to optimize growth and skeletal outcomes in clinical practice."

Journal • Real-world evidence • Retrospective data • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult) • Growth Hormone Deficiency (Pediatric) • Pediatrics • IGF1

A Study to Learn About Somatrogon in Patients With Pediatric Growth Hormone Deficiency (p GHD) in India. (clinicaltrials.gov) - P=N/A | N=50 | Recruiting | Sponsor: Pfizer | Trial completion date: Dec 2026 ➔ Sep 2028

Trial completion date • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Pediatric) • Pediatrics

Budget impact analysis of Skytrofa for the treatment of pediatric growth hormone deficiency in a US health plan. (PubMed, J Med Econ) - "This analysis included daily somatropin, and weekly somatrogon and somapacitan, as comparators. The primary drivers of the cost reduction were decreased acquisition costs for daily somatropin and the elimination of wastage for weekly GHs. The inclusion of lonapegsomatropin in the formulary of a US health plan can result in a significant reduction in total costs for the plan."

HEOR • Journal • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Pediatric) • Pediatrics

Lipoatrophy following weekly growth hormone therapy: a case report. (PubMed, J Pediatr Endocrinol Metab) - "To our knowledge, this is only the second reported case of somatrogon-induced lipoatrophy in the literature. This case also showed that lipoatrophy developing as a result of somatogron treatment is a reversible side effect."

Journal • Lipodystrophy

First 100 patients receiving long-acting growth hormone therapy: real-world evaluation from INSIGHTS-GHT registry. (PubMed, Orphanet J Rare Dis) - "Following the market launch of three LAGH products in Germany (lonapegsomatropin, somapacitan, and somatrogon) we aimed to provide early real-world evidence on their use in order to obtain an initial picture on patient selection and physician preferences outside of clinical trials.We report in this interim analysis on 70 pediatric patients from 15 centers across Germany as well as 31 adult patients from 6 German centers under LAGH treatment. More than half (55%) received the LAGH starting dose according to the manufacturer's recommendation, while 41% began with a lower-than-recommended dose. Our findings provide early insights into LAGH therapy adoption and highlight the need for continued follow-up to evaluate long-term efficacy, adherence, and safety in real-world settings."

HEOR • Journal • Real-world evidence • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult) • Growth Hormone Deficiency (Pediatric) • Pediatrics • IGF1 • IGFBP3

Comparative Efficacy of Once-Weekly Somatrogon Versus Daily Growth Hormone Therapy in Children With Idiopathic Growth Hormone Deficiency: A Real-World Retrospective Study From Greece. (PubMed, Cureus) - "Both treatments were well tolerated. Conclusion Both daily and weekly GH regimens significantly increased height and height SDS, with similar efficacy and safety profiles, over the six-month period."

Journal • Real-world evidence • Retrospective data • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Pediatric) • Pediatrics

Real world data for the first year of treatment with somatrogon of children and adolescents with growth hormone deficiency (GHD) (ESPE-ESE 2025) - "In this group of patients, treatment with somatrogon was well tolerated, proved to be efficient in accelerating growth velocity and improving height z-score. A trend of increasing BMI z-score was noticed that needs further observation. The limitation of this study is the small number of children and the short duration."

Clinical • Real-world • Real-world evidence • Dermatology • Endocrine Disorders • Growth Hormone Deficiency • Pain • IGF1

Should The Initial Dose Of Long-Acting Growth Hormone Therapy Somatrogon For Pediatric Growth Hormone Deficiency Be Standard For Each Child? (ESPE-ESE 2025) - "Somatrogon may be a suitable option in PGHD patients, but the recommended initial dose of 0.66 mg/kg/week may need to be individualized, particularly in children with severe GHD and in Tanner stage 4/5 of puberty. Careful monitoring and dose adjustment based on IGF-1 levels are necessary to maintain safety and efficacy."

Clinical • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Pediatric) • Pediatrics • IGF1

Efficacy and safety of once-weekly somatrogon in adults with growth hormone deficiency: a phase 3 study (ESPE-ESE 2025) - No abstract available

Clinical • P3 data • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult)

Self-resolving regional Lipodystrophy secondary to Somatrogon Therapy: A Case Report (ESPE-ESE 2025) - "This case highlights regional lipodystrophy as a potential side effect of somatrogon therapy in a child with GHD. Timely identification and intervention through instituting proper injection site rotation led to successful resolution, without discontinuing therapy. This highlights the importance of patient and caregiver education on injection techniques to optimize therapeutic outcomes and adherence."

Case report • Clinical • Endocrine Disorders • Growth Hormone Deficiency • Lipodystrophy • Metabolic Disorders • Pain

Efficacy and safety of once-weekly somatrogon in adults with growth hormone deficiency: a phase 3 study (ESPE-ESE 2025) - P3 | "Once-weekly somatrogon treatment significantly improved most body composition parameters and was well tolerated in adults with GHD."

Clinical • P3 data • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult) • Growth Hormone Deficiency (Pediatric) • Infectious Disease • Pain • Pediatrics • IGF1

Our experience with Somatrogon: Therapeutic efficacy, adult height, safety and adherence. (ESPE-ESE 2025) - "- Somatrogon (LAGH) has shown safety and efficacy in promoting linear growth in this patient´s group treated in our clinic. Similarly to daily GH in the treatment of GH deficiency; although with excellent adherence and less treatment burden. - Four patients achieved adult height near their target height."

Adherence • Clinical • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult) • IGF1 • IGFBP3

INSIGHTS-GHT: First evaluation of paediatric patients with long-acting growth hormone therapy (LAGH) from the German registry (ESPE-ESE 2025) - "In particular, since the end of 2023, patients treated with the newly approved long-acting GH preparations (LAGH) Ngenl"

Clinical • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult) • Growth Hormone Deficiency (Pediatric) • Pediatrics • Rare Diseases

Transitioning from Daily to Weekly Growth Hormone Therapy in Pediatric Idiopathic Growth Hormone Deficiency: Efficacy and Quality-of-Life Perspectives (ESPE-ESE 2025) - "Recently, a weekly GH treatment with NGENLA has become available in Greece, offering an alternative regimen... Transitioning from daily to weekly GH therapy appears to be a viable alternative for managing idiopathic GHD. The weekly regimen maintains growth efficacy while enhancing patient satisfaction and quality of life. Improved adherence and psychosocial benefits observed in this study suggest that weekly treatment with NGELA, can streamline everyday routines for patients and families, potentially leading to better long-term treatment outcomes."

Clinical • HEOR • Late-breaking abstract • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Pediatric) • Mood Disorders • Pain • Pediatrics • Psychiatry • IGF1

Comparison of the Effects of Weekly and Daily Treatment on Auxology, Metabolism and Quality of Life in Cases with Growth Hormone Deficiency (ESPE-ESE 2025) - "The efficacy, tolerability and quality of life of once-weekly somatrogon were likely to those of once-daily somatropin for GHD. Note: The study is still ongoing and a preliminary analysis has been conducted based on 41 patients with completed 6-month data. The full dataset is expected to be completed by the time of the conference."

Clinical • HEOR • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Pediatric) • Pediatrics

Withdrawal of applications (European Medicines Agency) - "An application for an initial marketing authorisation was withdrawn. Dazluma (troriluzole hydrochloride monohydrate) was intended for the treatment of spinocerebellar ataxia genotype 3 (SCA3), an inherited brain disorder that affects coordination and balance....An application to extend the therapeutic indication of Ngenla (somatrogon) in the treatment of adults with growth hormone deficiency was also withdrawn."

CHMP • Ataxia • Growth Hormone Deficiency (Adult)

Detection of the GH analogue somatrogon in doping control urine samples by means of LC-HRMS/MS. (PubMed, Sci Rep) - "Consequently, anti-doping laboratories can also use urine to confirm the presence of somatrogon in athletes, where the GH differential isoform assay yielded atypical results in the corresponding serum specimens. Moreover, the latter assay also proved to be valuable as screening tool for the identification of urine samples potentially containing the GH analog."

Journal

Long-Acting Growth Hormone for Pediatric Growth Hormone Deficiency. (PubMed, J Endocr Soc) - "Five trials were eligible for inclusion in a Bayesian network meta-analysis; 3 contributed to the base case network, including 3 LAGHs (lonapegsomatropin, somapacitan, and somatrogon) and daily somatropin. In this network meta-analysis, lonapegsomatropin was the only LAGH associated with better growth outcomes. No significant differences were detected regarding SAEs; other safety outcomes could not be analyzed."

Journal • Review • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Pediatric) • Pediatrics • IGF1

Long-acting growth hormone in the treatment of children with growth hormone deficiency. (PubMed, Expert Rev Endocrinol Metab) - "The review focuses on the LAGH approved from both the Food and Drug Administration (FDA) and European Medicines Agency (EMA) for the treatment of pediatric growth hormone deficiency (PGHD): Lonapegsomatropin, Somatrogon and Somapacitan. Even if current evidence suggests a non-inferiority of all the three LAGH formulations when compared to daily rhGH, long-term concerns persist regarding the non-physiological GH profile associated with LAGH, characterized by weekly instead of daily peaks. Further research and real-life studies are required to better define the long-term efficacy of these formulations."

Journal • Review • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult) • Growth Hormone Deficiency (Pediatric) • Pediatrics