In Development for the Diagnosis of Childhood-Onset
Growth Hormone Deficiency
Macimorelin, a ghrelin receptor agonist, is a small molecule that stimulates the secretion of growth hormone (GH) from the pituitary gland. Stimulated growth hormone levels are measured in blood samples after oral administration of macimorelin for the assessment of GHD.
Macimorelin, is the only U.S. FDA and European Commission approved oral test indicated for the diagnosis of adult growth hormone deficiency (AGHD).
We are also leveraging the clinical success and compelling safety profile of macimorelin to develop it for the diagnosis of childhood-onset growth hormone deficiency (CGHD), an area of significant unmet need.
Aeterna is regaining rights to macimorelin (Macrilen™) in the United States and Canada in May 2023 and is engaged in robust business development efforts to identify and secure a new development and commercialization partner in these territories.
“Because the macimorelin test is simple, well tolerated with minimal side effects, and of shorter duration with only 3 to 4 blood draws compared to other GH–stimulation tests, it is anticipated that its use will increase over time.”*
“Very promising test that is easy to conduct with high reproducibility, safety, and diagnostic accuracy comparable to the ITT and GHRH plus ARG test.”*
AACE 2019 Guidelines (https://pubmed.ncbi.nlm.nih.gov/31760824/).
*AACE 2019 Guidelines: American Association Of Clinical Endocrinologists And American College Of Endocrinology Guidelines For Management Of Growth Hormone Deficiency In Adults And Patients Transitioning From Pediatric To Adult Care, 2019
American Association of Clinical Endocrinologists 2019 Guidelines
Childhood-Onset Growth Hormone Deficiency (CGHD)
Growth Hormone is Critical to Child Development
Childhood-onset growth hormone deficiency (CGHD) is an endocrine condition associated with a broad range of health issues from childhood through to adulthood, which requires particular attention during the transition period from adolescence to young adulthood.
Reduction in auxological parameters:
• Short stature
• Low growth velocity (speed) for age
• Increased fat around the waist
• Delayed tooth development
Number of Potential Tests Annually
*Navigant study 2009, TVG study 2017, Huron Consulting study 2018
GHD in children can be congenital or acquired and either isolated or combined with other pituitary hormone defects. There are many well-defined causes of GHD in children, but the cause is often unknown (idiopathic GHD). The definitions of the various types of GHD and other causes of short stature are given in the International Classification of Pediatric Endocrine Diagnoses (ICPED).
Childhood-onset growth hormone deficiency and the transition to adulthood: current perspective