Aeterna Zentaris is dedicated to serving the unmet medical needs of people with rare endocrine diseases.
Aeterna Zentaris has extensive knowledge about Adult Growth Hormone Deficiency (AGHD) acquired through our development of macimorelin, an oral growth hormone secretagogue receptor agonist for the diagnosis of AGHD. As part of our clinical development strategy, we have fostered relationships worldwide at leading institutions and pituitary centers with renowned key opinion leaders including researchers, practicing endocrinologists, and nurse practitioners. We are in relentless pursuit of relieving the burden for endocrinologists and patients worldwide to reliably and accurately diagnosis AGHD. We are dedicated to leveraging our global endocrinology expertise acquired through the clinical development of macimorelin to establish a value-driven orphan disease portfolio in endocrinology through acquisition or in/out-licensing activities.
Macimorelin stimulates the secretion of growth hormone from the pituitary gland into the circulatory system. Clinical studies have demonstrated that growth hormone stimulation testing for AGHD with oral macimorelin is reliable, well-tolerated, reproducible, safe and a much simpler test to conduct than currently available options.
AGHD may occur in an adult subject who has a history of childhood onset Growth Hormone Deficiency or may occur during adulthood as an acquired condition.
About 6,000 cases of AGHD are reported each year in the United States, with an estimated 50,000 diagnosed adults. The prevalence after traumatic brain injury is estimated at 12% 1 . In general, the prevalence and incidence of AGHD is not well investigated in Europe by epidemiological studies. However, the NHS has estimated the number of subjects with AGHD in England and Wales with 12,600 and the prevalence in UK with 1 case in 10,000 of the adult population 2 . The prevalence in France was estimated by Sassiolas et al. with 2,638 cases, based on a ratio of 4.6 cases per 100,000 inhabitants. In the same study, a disease incidence was reported with 1.2 new cases per 100,000 inhabitants 3 . Regal et al. published data for Spain with an incidence of 4.44 cases per 100,000 female inhabitants and of 4.08 for male inhabitants 4 . Figures for Denmark taken from a publication from Stochholm et al. show an incidence of 1.42 new cases per 100,000 for females and of 1.9 cases for males. Considering a total population of 510 million for the European countries (EU-28), about 51,000 adults are estimated to be diagnosed with Growth hormone (GH) 5 .
In other areas of the world, the incidence and prevalence of AGHD are not known, and awareness and diagnosis remains low. GH not only plays an important role in growth from childhood to adulthood, but also helps promote a hormonally-balanced health status. AGHD mostly results from damage to the pituitary gland, which can be caused by tumors, traumatic brain injury, brain infarction, radiation or infections. It is usually characterized by a reduction in bone mineral density, lean body mass, exercise capacity, and overall quality of life as well as an increased cardiovascular risk profile.
GH not only plays an important role in growth from childhood to adulthood, but also helps promote a hormonally-balanced health status. AGHD mostly results from damage to the pituitary gland. It is usually characterized by a reduction in bone mineral density, lean body mass, exercise capacity, and overall quality of life as well as an increase of cardiovascular risks.
1. 2016 Endocrine Society ‘Hormonal Replacement in Hypopituitarism in Adults’ Guideline
2. NHS Technology Appraisal 64, August 2003
3. Sassiolas et al., European Journal of Endocrinology, 1999, 595-600
4. Regal et al., Clinical Endocrinology, 2001, 735-740
5. Stochholm et al., European Journal of Endocrinology, 2006, 61-71
For more information about Macimorelin in the United States and Canada please visit www.macrilen.com.