Current understanding and perspectives on growth and long-acting GH therapy in Japan

From the perspective of clinical pediatric endocrinology, progress in molecular biology over the past few decades has improved our understanding of growth physiology. This progress was particularly conspicuous in the early era of Sanger-based sequencing and has continued into the current era of next-generation sequencing. The first half of this review summarizes the current understanding about growth. The latter half discusses the development of long-acting GH (LAGH), an important, recent topic in pediatric endocrinology, with a focus on the Japanese market. LAGH is administered weekly over several years in some developed countries as treatment for GH deficiency (GHD). In Japan, somatrogon and somapacitan are available, and lonapegsomatropin has completed a clinical trial. As per recent meta-analyses, these three products were not inferior to conventional, daily GH therapy for short-term growth in pediatric GHD. Although LAGH is promising, some concerns remain. Well-designed clinical research is needed to expand the indications for LAGH to other conditions, such as idiopathic short stature. Finally, future research on orally administered agents may open new avenues for the treatment of short stature.