Efficacy and Safety of Weekly Somatrogon vs Daily Somatropin in Children With Growth Hormone Deficiency: A Phase 3 Study

Deal, Cheri; Steelman, Joel; Vlachopapadopoulou, Elpis; Stawerska, Renata; Silverman, Lawrence; Phillip, Moshe; Kim, Ho-Seong; Ko, Cheol Woo; Malievsky, Oleg; Cara, José; Roland, Carl L.; Taylor, Carrie Turich; Valluri, Srinivas Rao; Wajnrajch, Michael; Pastrak, Aleksandra; Miller, Bradley S.

doi:10.1210/clinem/dgac220

Cited by 43 publications

(39 citation statements)

References 35 publications

(48 reference statements)

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“…No severe AEs, SAEs, or withdrawals due to AEs were reported for any treatment group in the Phase 2 study [ 15 ]. The incidence of severe TEAEs and serious AEs for the somatrogon group in the Phase 3 study was 8.3% and 2.8%, respectively, with 1 patient discontinued due to an AE [ 16 ].…”

Section: Discussionmentioning

confidence: 99%

“…Somatrogon is currently approved in Canada, Australia, Japan, the UK, and the EU as a once-weekly SC injection for the treatment of children with growth disturbance due to insufficient secretion of GH, also referred to as pediatric GHD. Results from a global Phase 3 study indicate that once-weekly somatrogon was generally well tolerated and demonstrated noninferiority to once-daily Somatropin in promoting growth in pediatric patients with GHD [ 16 ].…”

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confidence: 99%

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Treatment Burden of Weekly Somatrogon vs Daily Somatropin in Children With Growth Hormone Deficiency: A Randomized Study

Maniatis¹,

Carakushansky

Galcheva

et al. 2022

Journal of the Endocrine Society

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Context Somatrogon is a long-acting recombinant human growth hormone treatment developed as a once-weekly treatment for pediatric patients with growth hormone deficiency (GHD). Objective Evaluate patient and caregiver perceptions of the treatment burden associated with the once-weekly somatrogon injection regimen vs a once-daily Somatropin injection regimen. Methods Pediatric patients (≥3 to <18 years) with GHD receiving once-daily somatropin at enrollment were randomized 1:1 to Sequence 1 (12 weeks of once-daily Somatropin, then 12 weeks of once-weekly somatrogon) or Sequence 2 (12 weeks of once-weekly somatrogon, then 12 weeks of once-daily Somatropin). Treatment burden was assessed using validated questionnaires completed by patients and caregivers. The primary endpoint was the difference in mean overall life interference (LI) total scores after each 12-week treatment period (somatrogon vs Somatropin), as assessed by questionnaires. Results Of 87 patients randomized to Sequence 1 (n = 43) or 2 (n = 44), 85 completed the study. Once-weekly somatrogon had a significantly lower treatment burden than once-daily Somatropin, based on mean overall LI total scores after somatrogon (8.63) vs Somatropin (24.13) treatment (mean difference –15.49; 2-sided 95% CI –19.71, –11.27; P < .0001). Once-weekly somatrogon was associated with greater convenience, higher satisfaction with treatment experience, and less LI. The incidence of treatment-emergent adverse events (TEAEs) for Somatropin and somatrogon was 44.2% and 54.0%, respectively. No severe or serious AEs were reported. Conclusion In pediatric patients with GHD, once-weekly somatrogon had a lower treatment burden and was associated with a more favorable treatment experience than once-daily Somatropin.

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Section: Discussionmentioning

confidence: 99%

mentioning

confidence: 99%

Treatment Burden of Weekly Somatrogon vs Daily Somatropin in Children With Growth Hormone Deficiency: A Randomized Study

Maniatis¹,

Carakushansky

Galcheva

et al. 2022

Journal of the Endocrine Society

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“…As described earlier, the pharmacodynamic profiles of IGF-I levels following an injection of the different LAGH products show a significant increase of IGF-I from baseline to peak and with return to baseline before the next injection (11,12,15). It is likely that the efficacy of LAGH will correlate with the average IGF-I level achieved (32).…”

Section: Dose Adjustmentmentioning

confidence: 67%

“…In the phase III trial of somatrogon in PGHD, children receiving somatrogon 0.66 mg/kg once weekly had mean IGF-I SDS at week 52 of +0.65 SDS compared to -0.69 SDS in children receiving DGH ( Table 2 ) ( 11 ). There are no data available yet for IGF-I values in children treated with somatrogon for longer periods.…”

Section: Introductionmentioning

confidence: 99%

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What do we do now that the long-acting growth hormone is here?

Miller

2022

Front. Endocrinol.

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In standard 52-week phase III clinical trials, once weekly lonapegsomatropin, somatrogon and somapacitan have been found to yield non-inferior height velocities and similar safety profiles to daily GH (DGH) in children with pediatric growth hormone deficiency (PGHD).Lonapegsomatropin, a long-acting GH therapy (LAGH), was approved by the United States Food and Drug Administration (FDA) in August 2021 for the treatment of PGHD and has also been approved in other regions of the world. Somatrogon was approved for the treatment of PGHD beginning in some regions beginning in late 2021. Somapacitan was approved by the FDA for the treatment of Adult GHD in August 2020. The phase III clinical trial of somapacitan for the treatment of PGHD has been completed and demonstrated non-inferiority of somapacitan to DGH.New LAGH products may improve patient adherence, quality of life and clinical outcomes, particularly in patients with poor adherence to daily GH injections in the future. With the availability of new LAGH products, clinicians will need to identify the best candidates for LAGH therapy and understand how to monitor and adjust therapy. Long-term surveillance studies are needed to demonstrate adherence, efficacy, cost-effectiveness and safety of LAGH preparations and to understand how the non-physiological pharmacokinetic and pharmacodynamic profiles following administration of each LAGH product relate to short- and long-term safety and efficacy of LAGH therapy.

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A Randomized, Cross‐Over Study Investigating the Comparability of Somatrogon‐ghla in 2 Different Drug Product Presentations

Manners,

Korth‐Bradley,

Wajnrajch

2024

Clinical Pharm in Drug Dev

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Somatrogon‐ghla is a long‐acting, recombinant human growth hormone approved for the treatment of pediatric patients with growth hormone deficiency. Forty‐nine healthy, adult males were enrolled in a randomized, crossover study to compare somatrogon exposure after subcutaneous doses administered using a frozen vial presentation or a prefilled, multiple dose pen. Somatrogon, insulin‐like growth factor‐I, and IGF‐1 binding protein‐3 concentrations were collected for up to 240 hours post dose to assess pharmacokinetic and pharmacodynamic responses. There was a 2‐week washout between administration of the doses. Seven participants did not complete the study due to withdrawal of consent (n = 2) or loss to follow‐up. Two treatment‐emergent adverse events, headaches, were judged by the investigator as possibly related to study drug administration. Both were mild. Injection site reactions were observed in 6/48 participants after administration with the pen and 12/46 after administration using the vial. Drug and biomarker concentrations were assessed using validated assays and noncompartmental methods were used to determine pharmacokinetic and pharmacodynamic parameters. Bioequivalence was demonstrated for somatrogon area under the concentration‐time curve, but not for the peak somatrogon concentration, where the lower limit of the 90% confidence interval for the ratio of pen/vial was 74.2%, which is less than the lower limit, 80.0%, dictated by bioequivalence criteria. The IGF‐1 responses were largely within bioequivalence limits. It was concluded that the 2 formulations are comparable.

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Efficacy and Safety of Weekly Somatrogon vs Daily Somatropin in Children With Growth Hormone Deficiency: A Phase 3 Study

Cited by 43 publications

References 35 publications

Treatment Burden of Weekly Somatrogon vs Daily Somatropin in Children With Growth Hormone Deficiency: A Randomized Study

Treatment Burden of Weekly Somatrogon vs Daily Somatropin in Children With Growth Hormone Deficiency: A Randomized Study

What do we do now that the long-acting growth hormone is here?

A Randomized, Cross‐Over Study Investigating the Comparability of Somatrogon‐ghla in 2 Different Drug Product Presentations

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