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

Withdrawal of applications (European Medicines Agency) - "An application for an initial marketing authorisation was withdrawn.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the therapeutic indication of Ngenla (somatrogon) in the treatment of adults with growth hormone deficiency was also withdrawn."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

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)

Real world data for the first year of treatment with somatrogon of children and adolescents with growth hormone deficiency (GHD) (ESPE-ESE 2025) - No abstract available

Clinical • Real-world • Real-world evidence • Endocrine Disorders • Growth Hormone Deficiency

Should The Initial Dose Of Long-Acting Growth Hormone Therapy Somatrogon For Pediatric Growth Hormone Deficiency Be Standard For Each Child? (ESPE-ESE 2025) - No abstract available

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

Self-resolving regional Lipodystrophy secondary to Somatrogon Therapy: A Case Report (ESPE-ESE 2025) - No abstract available

Case report • Clinical • Lipodystrophy • Metabolic Disorders

Our experience with Somatrogon: Therapeutic efficacy, adult height, safety and adherence. (ESPE-ESE 2025) - No abstract available

Adherence • Clinical

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)

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

Severe oedema during treatment with long acting GH (ESPE 2024) - "For practicability a treatment with long acting GH (Ngenla) 46 mg/week was started...We stopped the long acting GH-therapy and started furosemide treatment over 6 days which abolished the fluid retention...Thus, signs of fluid retention should be closely monitored during the treatment. Since serum sodium levels do not reflect the phenomenon, body weight, tubular reabsorption of sodium and body composition should considered as parameters."

Late-breaking abstract • Severe Hypoglycemia • IGF1

A Novel Case of Lipoatrophy Due to a Non-Pegylated Long-Acting Growth Hormone Somatrogon (ESPE 2024) - "After growing only 3 cm in a year, a clonidine test showed a peak value of 8.6 ng/ml, with an IGF-1 low normal level for her age (75 ng/ ml). Our case with somatrogon shows that lipoatrophy can also occur with a non-pegylated rhGH treatment rapidly within 11 weeks, likely due to repeated injections at the same site. Our findings highlight the importance of close monitoring and rotating injection sites during non-pegylated rhGH treatment, specifically with somatrogon, to reduce the risk of lipoatrophy."

Clinical • Late-breaking abstract • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Pediatric) • Inflammation • Lipodystrophy • Pediatrics • IGF1 • LPL

Tailoring the long-acting GH therapy in the real life (ESPE 2024) - "Considering the three switched patients, two completed 6 months of therapy while one 3 months. All showed an increase in IGF1 level compared to dGH, with values after the first month slightly higher in 4th than in 2nd day. The two patients with longer follow-up had IGF1 slightly above +2SDS for pubertal stage, remaining the same both at 3- and 6-month check-up."

Clinical • Endocrine Disorders • Pediatrics • IGF1

Successful Treatment of increasing chronic Hypoglycaemia by long acting GH a 15y old girl with reduced GH Secretion (ESPE 2024) - "Since 2 injections of GH/d were necessary to prevent hypoglycaemia, we started with long acting GH (Ngenla) 46 mg/week...The patient presents an unusual symptom of GH deficiency at this age. Continuous increase of GH levels with a long acting GH preparation abolished the symptoms."

Growth Hormone Deficiency • Hypoglycemia