TransCon human growth hormone for children with growth hormone deficiency: a technology evaluation

ABSTRACT

Introduction

Standard-of-care treatment for growth hormone deficiency consists of daily subcutaneous injections of recombinant human growth hormone, also known as somatropin. Although somatropin treatment is well established, the burden of daily injections can lead to poor adherence and quality of life. In this regard, a TransCon human growth hormone (lonapegsomatropin-tcgd) technology was developed to optimize the therapeutic effect of daily somatropin for the treatment of children with growth hormone deficiency.

Areas covered

The authors reviewed the effects of lonapegsomatropin-tcgd in children with growth hormone deficiency.

Expert opinion

Lonapegsomatropin-tcgd was found to be non-inferior to and superior to daily somatropin for annualized height velocity. In addition, the safety was comparable between them. As a result, the convenient dosing of lonapegsomatropin-tcgd has the potential to improve patient adherence, leading to increased efficacy and quality of life. Medication adherence, quality of life, long-term safety, and cost-effectiveness studies comparing lonapegsomatropin-tcgd and daily somatropin are required to confirm these possible benefits.

KEYWORDS:

• Efficacy
• growth hormone deficiency
• lonapegsomatropin-tcgd
• safety
• somatropin
• transcon technology

Article highlights

• A TransCon technology human growth hormone was developed (lonapegsomatropin-tcgd) for the treatment of children with growth hormone deficiency.

• Lonapegsomatropin-tcgd was found to be non-inferior to and superior to daily somatropin for annualized height velocity. The safety was comparable between them.

• The convenient dosing of lonapegsomatropin-tcgd may improve patient adherence, leading to increased efficacy and quality of life.

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

A reviewer on this manuscript has disclosed that they are a Principal Investigator for Asecndis sponsored foresiGHt trial. Another reviewer on this manuscript has disclosed that they have received honoraria and research grants funds from NovoNordisk who produce the long-acting GH somapacitan. Peer reviewers on this manuscript have no other relevant financial relationships or otherwise to disclose.

Additional information

Funding

This paper was not funded.