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

Immunogenicity Profile of Lonapegsomatropin During Long-Term Treatment in Children With Growth Hormone Deficiency. (PubMed, AAPS J) - "These findings support the long-term low immunogenicity of lonapegsomatropin in children with GHD, consistent with somatropin. Importantly, the multi-assay tiered strategy applied here was robust and reproducible, enabling sensitive detection of ADAs against the intact prodrug, mPEG, and released somatropin across trials, and demonstrated its clinical applicability for evaluating a complex long-acting growth hormone like lonapegsomatropin."

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

Treatment of growth hormone deficiency with long-acting growth hormones in children, adolescents and adults: Clinical evidence and practical considerations. (PubMed, Ann Endocrinol (Paris)) - "Three once-weekly LAGH products are currently available in the EU: lonapegsomatropin, somatrogon and somapacitan. With a focus on somapacitan, currently the only LAGH approved in France for both children and adults, the review discusses practical aspects of dosing and monitoring across age groups and addresses use in special populations and during the transition period from paediatric to adult care. Ongoing follow-up and real-world studies will further clarify long-term safety, adherence and metabolic outcomes."

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

HighLiGHts Phase 3 Trial Design: Lonapegsomatropin in Children With Short Stature or Growth Failure Due to Turner Syndrome, SHOX Deficiency, Small for Gestational Age, or Idiopathic Short Stature (ESPE 2026) - No abstract available

Clinical • P3 data • Genetic Disorders • Idiopathic Short Stature • Small for Gestational Age • Turners Syndrome

Dual Therapy With Navepegritide and Lonapegsomatropin Demonstrated Sustained Efficacy and Safety in Children With Achondroplasia at Week 78 of the COACH Trial (ASBMR 2026) - No abstract available

Clinical • Late-breaking abstract • Genetic Disorders

Long-Acting Growth Hormone Versus Daily Growth Hormone for Growth Hormone Deficiency Patients: A Network Meta-Analysis of Clinical Trials. (PubMed, Endocrinol Diabetes Metab) - "Once-weekly LAGH provides an effective frontline alternative to daily acting growth hormone, without compromising overall linear growth or safety, while displaying no differences in overall treatment discontinuation. Selection should be based on the patients' clinical profiles, specifically considering higher localized reactions with Somatrogon. Clinicians should adhere to formulation-specific sampling windows (2-5 days post dose for Lonapegsomatropin; 96 h for Somatrogon) to accurately monitor steady-state IGF-1 levels."

Clinical • Journal • Retrospective data • Review • Dermatology • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Pediatric) • Pain • Pediatrics • IGF1

Diabetic ketoacidosis induced by long-acting growth hormone in a child without preexisting diabetes. (PubMed, JCEM Case Rep) - "A 13-year-old boy with obesity, normal fasting glucose, and hypopituitarism receiving levothyroxine, hydrocortisone, and desmopressin replacement therapies following treatment of a suprasellar nongerminomatous germ cell tumor initiated treatment with lonapegsomatropin at 0.23 mg/kg/week. This case suggests that acute LAGH-induced insulin resistance, potentially compounded by glucocorticoids and obesity, may precipitate transient DKA even in patients without prior glucose abnormalities. Close glucose monitoring should be considered when initiating LAGH therapy in high-risk patients."

Journal • Diabetes • Endocrine Disorders • Genetic Disorders • Germ Cell Tumors • Growth Hormone Deficiency • Metabolic Disorders • Obesity • Oncology • Type 1 Diabetes Mellitus

Comparison of efficacy and safety between long-acting growth hormone and short-acting growth hormone in isolated growth hormone deficiency: a systematic review and meta-analysis of 16 randomized controlled trials involving 2,435 pediatric patients. (PubMed, Front Pediatr) - "Subgroup analyses were performed based on LAGH platform (PEG-LAGH, somatrogon, somapacitan, lonapegsomatropin) and treatment duration. Among children with isolated GHD, LAGH represents the preferred platform, offering both superior first-year height velocity and acceptable safety. In contrast, newer prodrug or albumin-binding LAGH formulations provide injection convenience but lack superior growth efficacy."

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

Safety and Effectiveness of Lonapegsomatropin in the Skybright Registry: Analysis of the First 100 Patients Enrolled with 2 Years of Follow-Up (ENDO 2026) - "IGF-1 SDS was maintained between 0 and +2.0 SDS, consistent with adequate GH replacement. Safety and efficacy outcomes were comparable to prior clinical trial results, supporting the effectiveness of lonapegsomatropin administered once weekly as part of routine clinical care for children and adolescents with GHD."

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

Navepegritide Combined with Lonapegsomatropin for the Treatment of Achondroplasia: An Open-Label Phase 2 Trial (ENDO 2026) - P2/3 | "One year of once weekly navepegritide and lonapegsomatropin were well tolerated and augmented proportionate growth in children with ACH, suggesting that lonapegsomatropin, through a distinct mode of action, complements navepegritide's therapeutic effects in children with ACH."

Clinical • P2 data • Genetic Disorders • FGFR3

Patient-Centric Design of the Lonapegsomatropin Auto-Injector for Growth Hormone Deficiency: Usability Validation of Safe and Effective Use. (PubMed, Med Devices (Auckl)) - "Use deviations observed for injection tasks for all participant groups were low: patients (3.2% of tasks), caregivers (2.4%), and HCPs (2.8%). The user-centric design of the lonapegsomatropin Auto-Injector enables successful and confident use to deliver an injection as intended, with the potential to overcome known barriers associated with GH injections and facilitate adherence, which may improve treatment outcomes."

Journal • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Pediatric) • Mood Disorders • Pediatrics • Psychiatry

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=66 | 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

Navepegritide Combined with Lonapegsomatropin for the Treatment of Children with Achondroplasia: 52-Week Results from the Phase 2 COACH Trial. (PubMed, Eur J Endocrinol) - P2/3 | "In this prospective trial, the robust growth and anthropometric results suggest that by continuously relieving the pre-hypertrophic block in achondroplasia, navepegritide may make the growth plate more responsive to the strong complementary effect of lonapegsomatropin."

Journal • P2 data • Genetic Disorders

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=66 | Recruiting | Sponsor: Ascendis Pharma Endocrinology Division A/S | Not yet recruiting ➔ Recruiting

Enrollment open • 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)

Long-term Safety and Efficacy of Lonapegsomatropin in Adults With Growth Hormone Deficiency: Results From a 52-Week Open-Label Extension Trial (AACE 2026) - "Lonapegsomatropin treatment was well tolerated, with a safety profile that was consistent with treatment during the foresiGHt trial, with no new safety signals identified. Body composition parameters were generally maintained (in participants who continued"

Clinical • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult) • Infectious Disease • Respiratory Diseases

Facts and news about growth hormone replacement therapy in adults. (PubMed, Ann Endocrinol (Paris)) - "Traditional stimulation tests, such as the insulin tolerance and glucagon tests, are gold-standards for diagnosis, but the oral macimorelin test offers a safe, reliable and well-tolerated alternative...Long-acting growth hormone (LAGH) formulations, such as somapacitan, lonapegsomatropin and somatrogon, now enable weekly administration, improving adherence while maintaining efficacy and safety comparable to daily therapy. Early evidence suggests enhanced patient satisfaction and similar metabolic outcomes. Ongoing longitudinal studies are essential to clarify long-term safety."

Journal • Review • Cardiovascular • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult) • Musculoskeletal Diseases • Obesity • IGF1

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

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)