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somatropin

✓ Approved

USV Limited · GHR · 多肽类

什么是 somatropin？

somatropin 是一种多肽类，由USV Limited研发。该药已获批，用于治疗相关适应症，给药途径：Injectable (Others)。

药物档案

公司USV Limited
药物类别多肽类
分子靶点GHR
给药途径Injectable (Others)
状态Approved
来源： ClinicalTrials.gov, USV Limited

作用机制

分子靶点

somatropin 作用于 1 个分子靶点：

GHRgrowth hormone receptor (GHBP, GHIP)
需要更深入的分析？Noah AI 可解释复杂机制并与同类药物比较。

治疗适应症

somatropin 针对 1 个适应症，涉及 1 个治疗领域。

治疗领域疾病/病症分期
Endocrine disordersGrowth hormone deficiency✓ Approved

相关研究文献

PubMedThe Journal of clinical endocrinology and metabolism2026-10-26

Efficacy and Safety of Lonapegsomatropin in Children With Turner Syndrome: 52-Week Results From the New InsiGHTS Trial.

Davis Shanlee M SM, Abuzzahab M Jennifer MJ, Tryggestad Jeanie B JB, Fechner Patricia Y PY et al.

Treatment with growth hormone is effective in improving height outcomes in children with Turner syndrome. However, poor adherence-often influenced by daily injection burden-may compromise treatment outcomes. Lonapegsomatropin is a prodrug, administered once weekly, that provides sustained release of somatropin. To evaluate the efficacy and safety of lonapegsomatropin in children with Turner syndrome. New InsiGHTS is a phase 2 dose-ranging trial (ClinicalTrials.gov: NCT05690386). This trial was conducted across 19 centers in the United States. This trial enrolled and dosed 49 prepubertal children with Turner syndrome. Randomization was 1:1:1:1 to receive once-weekly lonapegsomatropin (0.24, 0.30, or 0.36 mg hGH/kg/week) or daily somatropin (0.35 mg/kg/week). The primary efficacy endpoint was annualized height velocity (AHV) at week 26. Secondary efficacy endpoints included AHV at week 52 and change from baseline in height standard deviation score (SDS) at weeks 26 and 52. At week 26, AHV was comparable between lonapegsomatropin starting dose cohorts (range: 9.23 to 10.36 cm/year) and the somatropin cohort (10.86 cm/year). At week 52, AHV was comparable between pooled lonapegsomatropin cohorts (9.05 cm/year) and the somatropin cohort (9.04 cm/year), with mean gains in height SDS of 0.60 and 0.67, respectively. Lonapegsomatropin was well tolerated with a safety profile similar to somatropin. Across evaluated doses, lonapegsomatropin treatment demonstrated comparable efficacy, safety, and tolerability to somatropin after 1 year, supporting development of lonapegsomatropin as a once-weekly treatment option for short stature in children with Turner syndrome.

PMID 42929032
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PubMedAnnals of pediatric endocrinology & metabolism2026-10-11

Somatrogon versus somatropin in children with growth hormone deficiency: a real-world matched-cohort study.

Orman Burçe B, Kücükali Gülin Karacan GK, Keskin Melikşah M, Erdeve Şenay Savaş ŞS

Somatrogon is a once-weekly growth hormone (GH) analog approved for GH deficiency (GHD), but real-world head-to-head data are limited. Using a matched-cohort design with a protocol-defined IGF-1 sampling time, we compared first-year growth outcomes and safety of somatrogon and daily somatropin in children with GHD. We conducted a retrospective matched-cohort study in 30 children with GHD (15 per group) at a single center. Groups were matched by age (±1 year), sex, and pubertal stage. Annual height velocity (AHV), height standard deviation score (SDS) gain, insulin-like growth factor 1 (IGF-1) SDS, insulin-like growth factor binding protein 3 (IGFBP-3), bone age advancement, and metabolic parameters were assessed at 12 months. AHV was similar in both groups (8.96±1.67 vs 8.84±1.57 cm/year; p=0.841). Height SDS gain did not differ (0.60±0.36 vs 0.53±0.33; p=0.569). IGF-1 SDS was numerically higher in the somatrogon group (+0.59 vs -0.14; p=0.132) but remained within safe limits in all patients. Bone age advancement, glucose, and insulin levels were similar between groups. No adverse events or injection-site reactions were documented during follow-up. In this matched cohort, no significant differences in growth or safety outcomes were observed between somatrogon and somatropin at one year. Somatrogon appears to be a reasonable alternative to daily GH therapy in children with GHD.

PMID 42860242
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PubMedJournal of the Endocrine Society2026-10-11

Up to 5 years of weekly somatrogon in children with growth hormone deficiency: final data from the global phase 3 study.

Stawerska Renata R, Deal Cheri L CL, La Torre Daria D, Taylor Carrie T CT et al.

This open-label extension (OLE) study aimed to evaluate the long-term efficacy and safety of once-weekly somatrogon in pediatric patients with growth hormone deficiency (GHD) following up to 5 years of somatrogon treatment. Patients who had previously completed 12 months of treatment (somatrogon or somatropin) in a phase 3, randomized, active-controlled study (NCT02968004) were eligible to enroll into this OLE study; somatropin-treated patients were switched to somatrogon (0.66 mg/kg/week), and somatrogon-treated patients received somatrogon at their previous dose. A total of 212 patients entered the OLE. Mean annualized height velocity at the end of OLE Year 1 (Y1), Y2, Y3, and Y4 was 8.11, 7.91, 7.02, and 6.56 cm/year, respectively. Mean height SDS increased in each year of the OLE and approached 0 SDS by OLE Y4. Mean (SD) IGF-I SDS was positive and within the normal range throughout the OLE. The incidence of adverse events (AEs) was 53.8% to 72.3%, with most being mild to moderate. The incidence of serious AEs in each year was low (0-4.7%), and none were related to treatment. The most common treatment-related AEs were injection-site pain and injection-site erythema. The presence of antidrug antibodies did not appear to impact safety or efficacy outcomes. Based on the final data from the largest cohort of somatrogon-treated patients to date, once-weekly somatrogon demonstrated sustained efficacy and an acceptable safety profile during the OLE period. Once-weekly somatrogon constitutes a valuable long-term alternative to once-daily somatropin, offering potentially improved adherence and treatment outcomes.

PMID 42856902
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PubMedEndocrine practice : official journal of the American College of Endocrinology and the American Association of Clinical Endocrinologists2026-10-09

Long-term Safety and Efficacy of Once-Weekly Lonapegsomatropin in Adults With Growth Hormone Deficiency: Results From a 52-Week Open-Label Extension Trial.

Biller Beverly M K BMK, Gilis-Januszewska Aleksandra A, Doknic Mirjana M, Picó Antonio Miguel AM et al.

To evaluate long-term safety and efficacy of once-weekly lonapegsomatropin in adults with growth hormone (GH) deficiency. This 52-week, multicenter, open-label extension trial enrolled adults with GH deficiency who completed the 38-week phase 3 foresiGHt trial, in which participants received lonapegsomatropin, placebo, or somatropin.1 In the extension, all participants received lonapegsomatropin. Safety assessment included adverse events (AEs), laboratory values, vital signs, immunogenicity, 12-lead electrocardiograms, and fundoscopy. Body composition (trunk percent fat, trunk fat mass, visceral adipose tissue, total body lean mass, trunk lean mass, and appendicular lean mass) was measured by dual-energy X-ray absorptiometry, and patient-reported outcomes were evaluated. Following foresiGHt completion, 220 (88.7%) participants enrolled in the extension. Across foresiGHt and the extension, common lonapegsomatropin-associated AEs were nasopharyngitis, upper respiratory infection, and headache. Injection site reactions were infrequent (5.1%) and mild or moderate. After up to 90 weeks of treatment, body composition was maintained in participants continuing lonapegsomatropin, and improved relative to foresiGHt baseline in those initiating or switching to lonapegsomatropin. Clinically meaningful improvements in patient-reported outcomes from foresiGHt baseline were observed; 43.5%, 40.6%, and 35.8% of participants achieving a ≥10-point improvement in Treatment Related Impact Measure-Adult GH Deficiency (TRIM-AGHD) total score among those continuing, initiating, or switching to lonapegsomatropin, respectively. Once-weekly lonapegsomatropin was generally well tolerated and was associated with sustained improvements in body composition and patient-reported outcomes for up to 90 weeks. These findings extend prior evidence and support the long-term benefits of lonapegsomatropin in adults with GH deficiency, including those switching from somatropin.

PMID 42855131
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PubMedThe AAPS journal2026-09-22

Immunogenicity Profile of Lonapegsomatropin During Long-Term Treatment in Children With Growth Hormone Deficiency.

Mygind Per Holse PH, Weiser Amiee A, Fürst Camilla Melin CM, Komirenko Allison S AS et al.

Monitoring and evaluation of immunogenicity is important for biological therapeutics due to the potential impact of antibodies on safety and efficacy. A comprehensive immunogenicity assessment was conducted to support clinical development of lonapegsomatropin, a prodrug of somatropin, administered once weekly. Lonapegsomatropin is approved in the United States for pediatric and adult growth hormone deficiency (GHD), and in other countries for pediatric GHD. Lonapegsomatropin consists of somatropin, an inert methoxy polyethylene glycol carrier (mPEG), and a proprietary TransCon® linker that transiently binds somatropin to the carrier. Four dedicated immunoassays were developed, validated, and applied in a risk-based tiered approach to rigorously assess the immunogenicity of lonapegsomatropin in children with GHD. Samples collected from 298 children with GHD treated with lonapegsomatropin for up to six years were analyzed for anti-drug antibodies (ADAs) against lonapegsomatropin (intact prodrug), released somatropin, and mPEG. Treatment-emergent anti-lonapegsomatropin, anti-somatropin, and anti-mPEG binding antibodies were detected in 4.7%, 5.4%, and 0.7% of treated children, respectively. ADA responses were low titer, transiently detected, and primarily observed within the first year of treatment, and no neutralizing antibodies were detected. 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.

PMID 42768234
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PubMedJournal of comparative effectiveness research2026-09-10

Cost-effectiveness of a connected injection device for daily somatropin therapy in pediatric growth hormone deficiency in Spain: a scenario-based microsimulation analysis using real-world data.

Arriba Antonio de A, Roeder Claudia C, Nivelle Emilia E, Dommelen Paula van PV et al.

Aim: Growth hormone deficiency (GHD) in pediatric patients is characterized by impaired growth and reduced quality of life. Recombinant human growth hormone therapy requires consistent daily administration, yet adherence remains challenging. Electronic injection devices with adherence monitoring, like Easypod®, may support more consistent administration and enable more accurate assessment of treatment response. This study evaluated the cost-effectiveness of somatropin administered via a connected injection device (EasyPod) compared with nonconnected devices in pediatric patients with GHD in Spain. Materials & methods: A microsimulation model simulated 10,000 pediatric patients aged 2-13 years and incorporated adherence-dependent growth response, treatment discontinuation, costs and utilities. Outcomes included final height, height gain, cost per cm gained and incremental cost per quality-adjusted life year gained. Scenario and sensitivity analyses assessed the impact of parameter and structural uncertainty. Results: Easypod was associated with greater height gains than nonconnected devices. Projected final height at bone maturation was 163.04 cm with Easypod versus 159.21 cm with nonconnected devices, an incremental gain of 3.83 cm. Cost per cm gained was €2625 with Easypod and €3158 with nonconnected devices, a saving of €534 per cm. The incremental cost per quality-adjusted life year gained was €27,200 within Spain's willingness-to-pay threshold. Scenario and sensitivity analyses showed consistent results. Conclusion: In this model-based analysis, Easypod was associated with improved growth outcomes and was cost-effective compared with nonconnected devices in pediatric GHD patients in Spain. Continuous adherence monitoring may support treatment optimization and more efficient healthcare resource utilization.

PMID 42720214
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