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Lonapegsomatropin: Pediatric First Approval

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Abstract

Lonapegsomatropin (lonapegsomatropin-tcgd; SKYTROFA®), a long-acting prodrug of somatropin (human growth hormone), is in development by Ascendis Pharma as a treatment for growth hormone deficiency in pediatric and adult patients. Lonapegsomatropin received its first approval in August 2021 in the USA for the treatment of pediatric patients at least 1 year of age (and weighing ≥ 11.5 kg) with growth failure due to inadequate secretion of endogenous growth hormone. Lonapegsomatropin is administered as a once-weekly subcutaneous injection; the sustained release of somatropin from lonapegsomatropin eliminates the need for daily somatropin injections. This article summarizes the milestones in the development of lonapegsomatropin leading to this first pediatric approval for the treatment of growth hormone deficiency.

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References

  1. Ascendis Pharma. Ascendis Pharma A/S announces U.S. Food and Drug Administration approval of SKYTROFA(Rm) (lonapegsomatropin-tcgd), the first once-weekly treatment for pediatric growth hormone deficiency [media release]. 25 Aug 2021. http://www.ascendispharma.com.

  2. Yuen KCJ, Miller BS, Boguszewski CL, et al. Usefulness and potential pitfalls of long-acting growth hormone analogs. Front Endocrinol. 2021;12:637209.

    Article  Google Scholar 

  3. Gilfoyle D, Mortensen E, Christoffersen ED, et al. A first-in-man phase 1 trial for long-acting TransCon growth hormone. Growth Horm IGF Res. 2018;39:34–9.

    Article  CAS  PubMed  Google Scholar 

  4. Thornton PS, Maniatis AK, Aghajanova E, et al. Weekly lonapegsomatropin in treatment-naive children with growth hormone deficiency: the phase 3 heiGHt trial. J Clin Endocrinol Metab. 2021. https://doi.org/10.1210/clinem/dgab529.

    Article  PubMed  PubMed Central  Google Scholar 

  5. US Food and Drug Administration. SKYTROFA™ (lonapegsomatropin-tcgd): highlights of prescribing information. 2021. https://www.accessdata.fda.gov/. Accessed 12 Oct 2021.

  6. Silverman BL, Blethen SL, Reiter EO, et al. A long-acting human growth hormone (Nutropin Depot®): efficacy and safety following two years of treatment in children with growth hormone deficiency. J Pediatr Endocrinol Metab. 2002;15:715–22.

    Article  CAS  PubMed  Google Scholar 

  7. Fleseriu M, Jorgensen JO, Yuen KCJ, et al. Design of the ForesiGHt trial: a multicenter, randomized, placebo- and active-controlled trial to compare once-weekly TransCon hGH (lonapegsomatropin) to placebo and daily somatropin in adults with growth hormone deficiency (GHD) [abstract]. J Endocr Soc. 2021;5(Suppl 1):A519–20.

    Article  PubMed Central  Google Scholar 

  8. Höybye C, Pfeiffer AFH, Ferone D, et al. A phase 2 trial of long-acting TransCon growth hormone in adult GH deficiency. Endocr Connect. 2017;6(3):129–38.

    Article  PubMed  PubMed Central  Google Scholar 

  9. Beckert M, Gilfoyle D, Mikkelsen JM, et al. Design and clinical development of TransCon growth hormone for growth hormone deficiency (GHD) [abstract no. P1–P618 plus poster]. Growth Horm IGF Res. 2016;30–31(Suppl 1):S23–4.

  10. Ascendis Pharma. Ascendis Pharma announces formation of VISEN Pharmaceuticals to develop and commercialize TransCon(Tm) endocrinology rare disease therapies in China [media release]. 8 Nov 2018. http://www.ascendispharma.com.

  11. Ascendis Pharma. Ascendis Pharma Form 20-F December 2020. http://investors.ascendispharma.com/. Accessed 12 Oct 2021.

  12. Chatelain P, Malievskiy O, Radziuk K, et al. A randomized phase 2 study of long-acting TransCon GH vs daily GH in childhood GH deficiency. J Clin Endocrinol Metab. 2017;102(5):1673–82.

    Article  PubMed  Google Scholar 

  13. Maniatis AK, Nadgir U, Saenger P, et al. Phase 3 FliGHt trial: experience of switching from daily growth hormone therapy to once-weekly TransCon hGH in children with growth hormone deficiency [abstract]. J Endrocr Soc. 2020;4(Suppl 1):A488–9.

    Google Scholar 

  14. Maniatis A, Nadgir U, Casella S, et al. Treatment experience of children with growth hormone deficiency switching from daily growth hormone to once-weekly TransCon hGH: phase 3 fliGHt trial [abstract no. P-95]. Horm Res Paediatr. 2020;93(Suppl 1):66–7.

    Google Scholar 

  15. Maniatis A, Nadgir U, Hofman P, et al. Lonapegsomatropin (TransCon hGH) in children with growth hormone deficiency: efficacy and safety of up to 2 years of treatment [poster]. In: PES 2021 virtual annual meeting; 2021.

  16. Maniatis AK, Casella SJ, Nadgir UM, et al. Efficacy and safety of up to 2 years of treatment with TransCon hGH (lonapegsomatropin) in treatment-naive and treatment-experienced children with growth hormone deficiency [abstract no. P33-3 plus poster]. In: 103rd annual meeting of the Endocrine Society; 2021.

  17. Sävendahl L, Cooke R, Tidblad A, et al. Long-term mortality after childhood growth hormone treatment: the SAGhE cohort study. Lancet Diabetes Endocrinol. 2020;8(8):683–92.

    Article  PubMed  Google Scholar 

  18. Thornton P, Casella S, Maniatis A, et al. A novel GH auto-injector for once-weekly TransCon hGH [abstract P-96]. Horm Res Paediatr. 2020;93(Suppl 1):67.

    Google Scholar 

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Authors and Affiliations

  1. Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand

    Yvette N. Lamb

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  1. Yvette N. Lamb
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Correspondence to Yvette N. Lamb.

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The preparation of this review was not supported by any external funding.

Authorship and Conflict of interest

During the peer review process the manufacturer of the agent under review was offered an opportunity to comment on the article. Changes resulting from any comments received were made by the authors on the basis of scientific completeness and accuracy. Yvette Lamb is a salaried employee of Adis International Ltd/Springer Nature, and declares no relevant conflicts of interest. All authors contributed to the review and are responsible for the article content.

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This profile has been extracted and modified from the AdisInsight database. AdisInsight tracks drug development worldwide through the entire development process, from discovery, through pre-clinical and clinical studies to market launch and beyond.

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Lamb, Y.N. Lonapegsomatropin: Pediatric First Approval. Pediatr Drugs 24, 83–90 (2022). https://doi.org/10.1007/s40272-021-00478-8

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