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

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

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)...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

Results of the foresiGHt Trial Support the Efficacy and Safety of Once-Weekly Lonapegsomatropin in Adults with Growth Hormone Deficiency (GHD) (ESPE-ESE 2025) - No abstract available

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

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

Clinical outcomes of switching to lonapegsomatropin from somatropin for treatment of pediatric growth hormone deficiency. (PubMed, J Pediatr Endocrinol Metab) - "Lonapegsomatropin may be a favorable option to reduce injection burden for those with pediatric GHD, though the manufacturer's recommended starting dose of 0.24 mg/kg/week may require individualization. Careful monitoring and dose adjustment based on IGF-1 levels are necessary to maintain safety and efficacy."

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

A Trial to Investigate Long Term Efficacy and Safety of Lonapegsomatropin in Adults With Growth Hormone Deficiency (clinicaltrials.gov) - P3 | N=233 | Completed | Sponsor: Ascendis Pharma Endocrinology Division A/S | Active, not recruiting ➔ Completed

Trial completion • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult)

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 Trial to Investigate Different Doses of Lonapegsomatropin Compared to Somatropin in Individuals With Turner Syndrome (clinicaltrials.gov) - P2 | N=48 | Active, not recruiting | Sponsor: Ascendis Pharma Endocrinology Division A/S | Trial completion date: Jun 2026 ➔ Dec 2027

Trial completion date • Genetic Disorders • Turners Syndrome • IGF1

COACH: A Phase 2 Clinical Trial to Evaluate Efficacy, Safety, and Tolerability of Navepegritide in Combination With Lonapegsomatropin in Children With Achondroplasia (clinicaltrials.gov) - P2 | N=22 | Active, not recruiting | Sponsor: Ascendis Pharma Growth Disorders A/S | Recruiting ➔ Active, not recruiting

Combination therapy • Enrollment closed • Genetic Disorders

Efficacy and Safety of Somapacitan Relative to Somatrogon and Lonapegsomatropin in Pediatric Growth Hormone Deficiency: Systematic Literature Review and Network Meta-analysis. (PubMed, Adv Ther) - "No efficacy and safety differences were identified in comparisons of once weekly somapacitan versus somatrogon and lonapegsomatropin, as well as daily GH. All treatments were generally well tolerated, with the exception of observed injection site pain for somatrogon."

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

COACH: A Phase 2 Clinical Trial to Evaluate Efficacy, Safety, and Tolerability of Navepegritide in Combination With Lonapegsomatropin in Children With Achondroplasia (clinicaltrials.gov) - P2 | N=18 | Recruiting | Sponsor: Ascendis Pharma Growth Disorders A/S | Not yet recruiting ➔ Recruiting

Combination therapy • Enrollment open • Genetic Disorders

Ascendis to Spotlight Latest Updates for TransCon Rare Endocrinology Disease Portfolio at ENDO 2024 (GlobeNewswire) - "Ascendis Pharma A/S...today announced that it will host informational booths, events, and presentations showcasing use of its innovative TransCon technology in the areas of growth hormone deficiency (GHD), hypoparathyroidism, achondroplasia, and Turner syndrome during ENDO 2024, the annual meeting of the Endocrine Society being held June 1-4, 2024, in Boston...'we look forward to our first in-person opportunity to share data from foresiGHt, our pivotal trial of TransCon hGH in adults with GHD.'"

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

COACH: A Phase 2 Clinical Trial to Evaluate Efficacy, Safety, and Tolerability of Navepegritide in Combination With Lonapegsomatropin in Children With Achondroplasia (clinicaltrials.gov) - P2 | N=18 | Not yet recruiting | Sponsor: Ascendis Pharma Growth Disorders A/S

Combination therapy • New P2 trial • Genetic Disorders

Results Of The Foresight Trial Support The Efficacy And Safety Of Once-weekly Lonapegsomatropin In Adults With Growth Hormone Deficiency (ghd) (ENDO 2024) - "Abstracts presented at a news conference are embargoed until the date and time of the news conference. The Endocrine Society reserves the right to lift the embargo on specific abstracts that are selected for promotion prior to or during ENDO."

Clinical • Late-breaking abstract • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult) • IGF1

Baseline Demographics and Design of the SkybriGHt Registry Study. A US Multi-center, Prospective, Non-interventional, Long-term, Effectiveness and Safety Study of Children and Adolescents with Growth Hormone Deficiency Treated with Lonapegsomatropin (ENDO 2024) - "The SkybriGHt registry will evaluate the long-term safety, effectiveness, and treatment use patterns (including demographics), with lonapegsomatropin in clinical practice. This preliminary baseline data show that patients were predominantly male, consistent with prior treatment patterns of GHD in the United States. Description of baseline characteristics of additional enrolled patients will be presented.Unless otherwise noted, all abstracts presented at ENDO must not be released to the press or the public until the date and time of presentation."

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

A Trial to Investigate Different Doses of Lonapegsomatropin Compared to Somatropin in Individuals With Turner Syndrome (clinicaltrials.gov) - P2 | N=48 | Active, not recruiting | Sponsor: Ascendis Pharma Endocrinology Division A/S | Recruiting ➔ Active, not recruiting | Trial completion date: Oct 2025 ➔ Jun 2026 | Trial primary completion date: Oct 2025 ➔ Dec 2024

Enrollment closed • Trial completion date • Trial primary completion date • Genetic Disorders • Turners Syndrome • IGF1

Long-acting growth hormone in the treatment of growth hormone deficiency in children: a systematic literature review and network meta-analysis. (PubMed, Sci Rep) - "PEG-LAGH (risk ratio [RR]: 1.00, 95% CrI: 0.82, 1.2) reduced the risk of adverse events compared with other LAGH (somatrogon, RR: 1.1, 95% CrI: 0.98, 1.2; somapacitan, RR: 1.1, 95% CrI: 0.96, 1.4; lonapegsomatropin, RR, 1.1, 95% CrI: 0.91, 1.3) and was comparable with DGH. This is the first study to indirectly compare the LAGH thorough a network meta-analysis and provide evidence of the optimal efficacy of various LAGH specifically PEG-LAGH and acceptable safety profile in prepubertal children with GHD."

Journal • Retrospective data • Review • Endocrine Disorders • Growth Hormone Deficiency

Developments in the Management of Growth Hormone Deficiency: Clinical Utility of Somapacitan. (PubMed, Drug Des Devel Ther) - "Other LAGHs, such as somatrogon (Ngenla®) (Pfizer, New York, United States) and lonapegsomatropin/TransCon GH (Skytrofa®) (Ascendis Pharma, Copenhagen, Denmark), are also currently approved and available for the treatment of CGHD in several countries. Additionally, the administration of somapacitan and timing of measurement of serum insulin-like growth factor-I (IGF-I) levels are summarized. Information on administration, advice on missed doses, and clinical guidelines are discussed, as well as identifying which patients are suitable for somapacitan therapy, and how to monitor and adjust dosing whilst on therapy."

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

A Trial to Investigate Long Term Efficacy and Safety of Lonapegsomatropin in Adults With Growth Hormone Deficiency (clinicaltrials.gov) - P3 | N=232 | Active, not recruiting | Sponsor: Ascendis Pharma Endocrinology Division A/S | Enrolling by invitation ➔ Active, not recruiting

Enrollment closed • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult)

foresiGHt: A Trial to Compare the Efficacy and Safety of Once-weekly Lonapegsomatropin With Placebo and a Daily Somatropin Product in Adults With Growth Hormone Deficiency (clinicaltrials.gov) - P3 | N=264 | Completed | Sponsor: Ascendis Pharma Endocrinology Division A/S | Active, not recruiting ➔ Completed | Trial completion date: Dec 2024 ➔ Dec 2023

Trial completion • Trial completion date • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult) • IGF1

Somatrogon injection for the treatment of pediatric growth hormone deficiency with comparison to other LAGH products. (PubMed, Expert Rev Endocrinol Metab) - "Growth responses, pharmacodynamics and safety data are compared to other LAGH products, lonapegsomatropin and somapacitan, in Phase 3 trials in pediatric GHD. Clinicians will need to identify the best candidates for LAGH therapy and understand how to safely monitor and adjust therapy. Long-term surveillance studies are necessary to demonstrate adherence, efficacy, cost-effectiveness, and safety of LAGH preparations."

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