Pediatric Growth Hormone Deficiency (PGHD) refers to the growth deficiency caused by impaired production or secretion of human Growth Hormone (hGH). Children with GHD suffer from short stature and seriously affects their overall physical and mental health. Children with GHD affected growing up and developing if untreated1. Early discontinuation of GH treatment is associated with increased fat mass, decreased muscle mass, and decreased bone density2,3,4, and increased risk of cardiovascular disease5,6. A high incidence of psychiatric disorders, usually accompanied by poor life quality, is associated with adults who were GHD as children7.

Standard of care (SoC) for GHD is subcutaneous injection of daily hGH. Patients with GHD often undergo thousands of daily subcutaneous injections over many years of therapy, resulting in a high treatment burden, poor treatment compliance, and fail to achieve expected treatment goal8. One study has shown that as the number of missed injections per week increases, the growth improvement decreases(as illustrated in bellowing figure).

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TransCon hGH is the long-acting growth hormone prodrug using the innovative "Transient Conjugation" technology. The technology differs from the other long-acting growth hormone analogues with different technology and is designed to ensure the release of unmodified active human growth hormone (hGH) at a predictable rate over one week, resulting in the same tissue distribution as daily growth hormone therapy9.

The pivotal global phase 3 study in patients with childhood growth hormone deficiency was completed in the USA and Europe. The annual height velocity (AHV) for TransCon hGH was greater compared to a daily hGH (the treatment difference was 0.86 cm/year between TransCon hGH and daily hGH, p value is 0.0088), the safety and tolerability profile was comparable with daily hGH10 (as illustrated in bellowing figure).

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Currently, there is no long-acting growth hormone therapy approved in the US or Europe that has been applied to clinical practice11. TransCon hGH reduces 86% injection times compared to daily hGH*, which is expected to improve patients’ treatment compliance. Also, the TransCon hGH auto-injector is designed to significantly improve the patient experience, including a small needle (31G). The auto-injector is also being designed for auto data capture and enables integration with a connected healthcare system. TransCon hGH with room temperature stability will be convenient for patients and caregivers to store and carry.

Biologics License Application (BLA) filling to the U.S. Food and Drug Administration (FDA) expected in the first half of 2020. TransCon hGH China Phase 3 study initiated by the end of 2019 by VISEN Pharma.

*Note: TransCon hGH requires 52 injections per year; daily hGH requires 365 injections per year. The calculation formula is(365-52)/365=86%

Reference Boer H, et al. J Clin Endocrinol Metab. 1997;82(7):2032–2036.

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3.Colle M, et al. Horm Res. 1993;39(5-6):192-6.

4.Johannsson G, et al. J Endocrinol Invest. 1999;22(5 Suppl):41-6.

5.Leong GM, et al. Horm Res. 2003;60(Suppl 1):78–85.

6.Colao A, et al. J Clin Endocrinol Metab. 2002 Aug;87(8):3650-5.

7.Stabler B, et al. Anxiety. 1996;2(2):86–89.

8.Cutfield WS,et al. PLoS One. 2011;6(1):e16223.

9.Sprogøe K, et al. Endocr Connect. 2017;6(8):R171–R181.

10.ENDO2019: OR17-4.

11.Lal RA, et al. Arch Endocrinol Metab. 2019;63(6):601–607.