Skytrofa (lonapegsomatropin-tcgd) / Ascendis, Royalty, Teijin, Specialised Therap - LARVOL DELTA

Long-term Safety and Efficacy of Lonapegsomatropin in Adults With Growth Hormone Deficiency: Results From a 52-Week Open-Label Extension Trial (AACE 2026) - "This abstract reports the long-term safety and efficacy outcomes of treatment with lonapegsomatropin in adults with growth hormone deficiency in the phase 3 extension of the foresiGHt trial."

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

Patient and Caregiver Preferences for Once-Weekly Injection Devices for the Treatment of Growth Hormone Deficiency. (PubMed, J Endocr Soc) - "The profile similar to somapacitan-beco had the highest predicted choice probability (82%) compared with the profiles similar to lonapegsomatropin-tcgd (16%) and somatrogon-ghla (2%). Understanding patient and caregiver preferences for LA-GHA devices with less injection pain and greater ease of use highlights the value of shared decision-making, potentially leading to better treatment adherence and subsequent clinical outcomes."

Journal • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult) • Pain

Long-Acting Growth Hormone for Treating Growth Hormone Deficiency in Children: A Meta-Analysis of Randomized Controlled Trials Focusing on Changes in Body Mass Index. (PubMed, J Clin Endocrinol Metab) - "An increase in body mass index SD score is associated with the first year of LAGH use."

Journal • Retrospective data • Endocrine Disorders • Growth Hormone Deficiency

ASND0047: A Phase 3 Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children with Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders (clinicaltrialsregister.eu) - P2/3 | N=14 | Not yet recruiting | Sponsor: Ascendis Pharma Endocrinology Division A/S

New P2/3 trial • Pan tumor • Genetic Disorders • Idiopathic Short Stature • Small for Gestational Age • Turners Syndrome • IGF1

BUDGET IMPACT MODEL OF LONAPEGSOMATROPIN FOR THE TREATMENT OF ADULT GROWTH HORMONE DEFICIENCY IN THE UNITED STATES (ISPOR 2026) - "Lonapegsomatropin is budget‑neutral to budget‑favorable during the 5-year period investigated, when replacing somapacitan. These findings support the economic viability of expanding the indication for lonapegsomatropin to AGHD. Future work should incorporate net prices/rebates and treatment‑specific utilization linked to persistence/adherence to refine payer‑relevant estimates."

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

Once-weekly Lonapegsomatropin was Efficacious and Well Tolerated in Chinese Children with Growth Hormone Deficiency: Results from a Phase 3 Randomized Trial. (PubMed, Horm Res Paediatr) - P3 | "Weekly lonapegsomatropin demonstrated non-inferiority and superiority in efficacy compared to daily somatropin among treatment-naive Chinese children with GHD. The treatment groups showed comparable safety and tolerability profiles."

Journal • P3 data • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Pediatric) • Pediatrics • IGF1

Once-Weekly Lonapegsomatropin in Adults With Growth Hormone Deficiency: are we ready to use it? (PubMed, J Clin Endocrinol Metab) - No abstract available

Journal • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult)

VISEN Pharmaceuticals has received approval from China’s National Medical Products Administration for its biologics license application for lonapegsomatropin (SKYTROFA), a once-weekly growth hormone replacement therapy for children and adolescents aged three and above with growth hormone deficiency. (TipRanks) - "The decision, supported by positive Phase 3 trial data in China showing superior efficacy and comparable safety versus daily human growth hormone, positions lonapegsomatropin as the only long-acting growth hormone in China that delivers unmodified human growth hormone and may improve treatment compliance."

China approval • Growth Hormone Deficiency

Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children and Adolescents With Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders (clinicaltrials.gov) - P3 | N=186 | Recruiting | Sponsor: Ascendis Pharma A/S | Not yet recruiting ➔ Recruiting

Enrollment open • Pan tumor • Genetic Disorders • Idiopathic Short Stature • Small for Gestational Age • Turners Syndrome • IGF1

Impact of growth hormone treatment on a 12-year-old female with newly diagnosed panhypopituitarism and distal arthrogryposis. (PubMed, Endocrinol Diabetes Metab Case Rep) - "Hydrocortisone (9 mg/m2/day) and levothyroxine (37.5 μg daily) were first initiated. Although GH was initially deferred due to concerns about worsening preexisting distal hand contractures, the family elected to begin weekly subcutaneous lonapegsomatropin-tcgd (Skytrofa, 7.6 mg, 0.24 mg/kg/week)...Distal arthrogryposis coexisting with GH deficiency did not worsen with GH therapy in this patient, supporting individualized risk-benefit analysis. Shared decision making is key when theoretical risks (e.g. joint symptoms) are weighed against the consequences of untreated GH deficiency."

Journal • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult) • Nephrology • Pediatrics • Renal Disease

Efficacy and Safety of Once-Weekly Lonapegsomatropin in Adults With Growth Hormone Deficiency: foresiGHt Trial Results. (PubMed, J Clin Endocrinol Metab) - P3 | "The foresiGHt trial met its primary efficacy endpoint by demonstrating superiority of lonapegsomatropin vs placebo with similar safety and tolerability, supporting its potential as a once-weekly treatment option for adults with GHD."

Journal • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult)

Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children and Adolescents With Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders (clinicaltrials.gov) - P3 | N=186 | Not yet recruiting | Sponsor: Ascendis Pharma A/S

New P3 trial • Pan tumor • Genetic Disorders • Idiopathic Short Stature • Small for Gestational Age • Turners Syndrome

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

Once-Weekly Lonapegsomatropin (TransCon hGH) Added to Once-Weekly Navepegritide (TransCon CNP) in Children with Achondroplasia: 26-Week Results from the Phase 2 COACH Trial (ASBMR 2025) - No abstract available

Clinical • Late-breaking abstract • P2 data • Genetic Disorders

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

New Long Acting Growth Hormone (Lonapegsomatropin) Induced Adverse Effects: A Pediatric Case Series (ENDO 2025) - "In our case series, reactions to newer weekly GH injections ranged from behavioral to systemic. Most adverse effects were mild and resolved after stopping the medication or with injection site rotation. To minimize the risk of lipoatrophy due to the lipolytic effect of GH, it is crucial to stress the importance of rotating the injection site."

Adverse events • Clinical • Dermatology • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Pediatric) • Immunology • Lipodystrophy • Pediatrics • Pruritus

Impact of Growth Hormone Treatment on a 12 Year Old Female with Newly Diagnosed Panhypopituitarism and Distal Arthrogryposis (ENDO 2025) - "Treatment began with hydrocortisone (9 mg/m^2/day) and levothyroxine (37.5 mcg daily) with normalization of thyroid hormone levels...She was started on weekly subcutaneous Skytrofa injections (7.6 mg SQ weekly, 0.24 mg/kg/week)...This case report highlights that initiation of GH therapy may lead to a significant growth improvement without aggravating arthrogryposis-related contractures. However, generalizations should be made with caution, and we emphasize the importance of shared-decision making and frequent follow up in the use of GH therapy in patients with congenital contractures."

Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult) • Nephrology • Renal Disease • Rheumatology • IGF1

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

Circulating IGF-1 Levels and Trends in Response to Daily and Weekly GH Therapies: A Comparative Analysis (ESPE-ESE 2025) - " Data were synthesized from eight studies analyzing IGF-1 response to various GH therapies, including daily rGH and weekly long-acting formulations such as somapacitan, PEG-rhGH, TransCon GH, and Nutropin Depot...Daily GH Therapies: Daily rGH therapy (e.g., Genotropin, Norditropin) resulted in more consistent IGF-1 levels with minor daily fluctuations, providing a steady profile throughout the week-(Lundberg et al., 2018)... Both weekly and daily GH therapies effectively elevate IGF-1 levels, with distinct advantages and limitations. Weekly formulations optimize patient adherence and long-term exposure, whereas daily rGH ensures consistent IGF-1 profiles. Table: IGF-1 Trends and Patterns Between Daily Vs Long-Acting-GH Therapies IGF-1 Response GH Therapy Type Key IGF-1 Trends IGF-1 Patterns Consistency Variable-Timing, Stable Higher consistency across Daily GH Fluctuates-Daily Long-Term days Dose-Dependent, Higher overall AUC due to Long-Acting GH Weekly-Peaks and..."

IGF1

Safety Profile of Large IGF-1 Fluctuations: Long-Acting Growth Hormone Therapy vs. Daily Growth Hormone Therapy (ESPE-ESE 2025) - "Material and A comprehensive review of studies was conducted comparing safety profiles between long-acting GH therapies (e.g., somapacitan, TransCon GH) and daily GH therapies (e.g., Genotropin). Long-acting GH therapy is a safe and effective alternative to daily GH therapy, despite larger IGF-1 fluctuations. Careful dose titration and regular monitoring are essential to minimize transient IGF-1 SDS excursions and maintain a favorable safety profile."

Clinical • Diabetes • Growth Hormone Deficiency • IGF1

Results of the foresiGHt Trial Support the Efficacy and Safety of Once-Weekly Lonapegsomatropin in Adults with Growth Hormone Deficiency (GHD) (ESPE-ESE 2025) - "Conclusions The results of the foresiGHt trial indicate that lonapegsomatropin is an efficacious and tolerable replacement for endogenous GH. Once-weekly dosing may be impactful for adults with GHD who typically manage multiple other medical therapies."

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

Usability and Preference Evaluation of the Somapacitan Pen-Injector and Lonapegsomatropin Autoinjector: Results of a US-Based Simulated-Use Study with Adolescent Patients and Caregivers. (PubMed, Patient Prefer Adherence) - "The somapacitan device was preferred over the lonapegsomatropin device among adolescents with GRDs and caregivers of individuals with GRDs. Individuals receiving GH treatment via the somapacitan device may be more likely to have higher treatment adherence than those using the lonapegsomatropin device."

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

Children with Growth Hormone Deficiency Treated With Lonapegsomatropin Demonstrated Sustained Height Improvements for up to 6 Years- enliGHten Trial Final Results. (PubMed, Horm Res Paediatr) - "Treatment of pediatric GHD with lonapegsomatropin in the enliGHten trial provided robust growth outcomes and maintained a safety profile comparable to daily GH in a population that included a broad range of pubertal status."

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