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

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

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 | Not yet recruiting ➔ Recruiting

Enrollment open • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Pediatric) • Pediatrics

Somatrogon in pediatric growth hormone deficiency: a comprehensive review of clinical trials and real-world considerations. (PubMed, Ann Pediatr Endocrinol Metab) - "The use of somatrogon is supported by phase II and III clinical trials demonstrating that once-weekly injections are noninferior to once-daily somatropin injections in terms of efficacy, safety, and tolerability and have the advantage of reduced treatment burden. This review summarizes the clinical trials of somatrogon and discusses the therapeutic profile and effects of treating pGHD with reduced injection frequency."

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

Commentary on "Somatrogon in pediatric growth hormone deficiency: a comprehensive review of clinical trials and real-world considerations". (PubMed, Ann Pediatr Endocrinol Metab) - No abstract available

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

Ngenla Subcutaneous Injection Special Investigation (clinicaltrials.gov) - P=N/A | N=1 | Active, not recruiting | Sponsor: Pfizer | Trial completion date: Apr 2028 ➔ Apr 2029 | Trial primary completion date: Apr 2028 ➔ Apr 2029

Trial completion date • Trial primary completion date • Endocrine Disorders • Growth Hormone Deficiency

IGF-1 Assessment During Weekly Somatrogon Treatment in Pediatric Patients With GH Deficiency. (PubMed, J Endocr Soc) - "These values were used to create a table showing the adjustments required to predict mean IGF-1 and IGF-1 SDS values depending on time after dose. We developed a simple method enabling physicians to predict mean weekly IGF-1 values using IGF-1 values measured at any point in the dosing interval."

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

Severe Lipoatrophy in a Growth Hormone Deficient Toddler Girl Treated with a Non-Pegylated Long-Acting Growth Hormone. (PubMed, Children (Basel)) - "The recurrence underscores the importance of proper injection techniques, particularly site rotation, in preventing localized adverse effects. Given the limitations of this case, where the recommended adjustments were not followed by the parents, it is crucial to emphasize that the administration of the preparation should be discontinued immediately upon the appearance of side effects such as lipoatrophy. Individual reactions to drugs are always possible, and this highlights the need for clinician vigilance in monitoring and addressing adverse effects promptly during treatments with LAGH."

Journal • Idiopathic Short Stature • Lipodystrophy

Long-acting growth hormones: innovations in treatment and guidance on patient selection in pediatric growth hormone deficiency. (PubMed, Ann Pediatr Endocrinol Metab) - "In contrast, LAGHs, such as somatrogon, somapacitan, and lonapegsomatropin, are designed for once-weekly administration, improving patient compliance and quality of life. Pediatric endocrinologists must carefully consider which patient groups would benefit most from this therapy, particularly those at risk for poor adherence to daily injections, such as patients undergoing multi-drug therapy, patients with needle phobia or behavioral disorders, very young children, adolescents, patients with separated parents, families that travel frequently, or children involved in activities like scouting. LAGHs present an opportunity to enhance therapeutic outcomes and adherence, but careful patient selection remains critical to maximizing their potential benefits."

Journal • Behavior Disorders • Endocrine Disorders • Genetic Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Pediatric) • Lipodystrophy • Mental Retardation • Pain • Pediatrics • Psychiatry

Current understanding and perspectives on growth and long-acting GH therapy in Japan. (PubMed, Clin Pediatr Endocrinol) - "In Japan, somatrogon and somapacitan are available, and lonapegsomatropin has completed a clinical trial. Well-designed clinical research is needed to expand the indications for LAGH to other conditions, such as idiopathic short stature. Finally, future research on orally administered agents may open new avenues for the treatment of short stature."

Journal • Review • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Pediatric) • Idiopathic Short Stature • Pediatrics

A Study to Learn About Somatrogon in Patients With Pediatric Growth Hormone Deficiency (p GHD) in India. (clinicaltrials.gov) - P=N/A | N=50 | Not yet recruiting | Sponsor: Pfizer | N=100 ➔ 50 | Trial completion date: Sep 2026 ➔ Dec 2026

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

Implementing a Bayesian approach using Stan with Torsten: Population pharmacokinetics analysis of somatrogon. (PubMed, CPT Pharmacometrics Syst Pharmacol) - "Stan took approximately 2.5 h total for the MCMC sampling on a high-performance computing platform (6 cores) and may be reduced further with additional computational resources. The model and comparisons presented show that with adequate computational resources, the Bayesian approaches using Stan and Torsten are useful for population PK analysis, especially for the analysis of special populations, small sample datasets, and when complex model structures are needed."

Journal • PK/PD data • Endocrine Disorders • Growth Hormone Deficiency

Real-world experience of using Long-Acting Growth Hormone Somatrogon in children and adolescents with growth hormone deficiency (ESPE 2024) - "To our knowledge, this is the largest and longest experience in the real world with Somatrogon. This treatment was found to be safe and effective, with a significant increase in height SDS despite a dose reduction."

Clinical • Real-world • Real-world evidence • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Pediatric) • Pediatrics • IGF1