Canada-based Aeterna Zentaris has filed a new drug application (NDA) to the US Food and Drug Administration (FDA) for its ghrelin agonist, macimorelin acetate (AEZS-130), a novel orally active small molecule.
The NDA is based on results from a Phase III trial, which showed that AEZS-130 has the potential to become the first orally-approved product that induces growth hormone release to assess adult growth hormone deficiency (AGHD), with accuracy compared to existing intravenous and intramuscular testing procedures.
Aeterna Zentaris president and CEO David Dodd said: "The submission of the NDA to the FDA is another major milestone in our strategy to bring macimorelin acetate to market as the first approved oral product for AGHD evaluation."
In addition, the company is also looking at potential expansion into traumatic brain injury victims and paediatrics, helping enhance its growth opportunities.
Macimorelin acetate, which received FDA orphan drug status, stimulates the secretion of growth hormone that plays a major role in growth from childhood to adulthood, as well as helps promote a hormonally-balanced health status.
Aeterna Zentaris owns the worldwide rights to this novel patented compound and has completed a Phase III trial for use in assessing AGHD, which affects around 75,000 adults across the US, Canada and Europe.
In addition, macimorelin acetate is in a Phase II trial as a treatment for cancer-induced cachexia.
AEZS-130 is a synthetic small molecule that is currently under development for the treatment for severe chronic diseases such as cancer induced cachexia and AGHD.
AGHD mostly results from damage to the pituitary gland and is usually characterised by a reduction in bone mineral density, lean mass, exercise capacity, and overall quality of life.
Image: Somatropin is a polypeptide hormone that stimulates growth and cell reproduction. Photo: courtesy of Wikipedia user POMaH Bekkep.
