Company plans to work with FDA in the coming months to prepare its NDA.
Aeterna Zentaris reported favorable top-line results of its completed Phase III study with AEZS-130 as the first oral diagnostic test for adult growth hormone deficiency (AGHD). The firm plans to meet with the FDA and work out the content of a submission for an NDA in the upcoming months.
“We believe that AEZS-130 could become the first approved oral test for the diagnosis of AGHD, providing patients with a potentially safer, accurate, and more convenient alternative to the current injectable tests,” says Juergen Engel, Ph.D., president and CEO at Aeterna Zentaris.
The primary efficacy parameters show that the study achieved both specificity and sensitivity at a level of 90% or greater. The use of AEZS-130 was shown to be safe and well tolerated overall throughout the completion of this trial, according to the company.
AEZS-130, a ghrelin agonist, is an orally active small molecule that stimulates the secretion of growth hormone. Since growth hormone secretion normally only occurs during sleep, a healthcare provider will measure how well the patient’s body responds to that stimulation based on the growth hormone levels over a period of time. Low growth hormone levels, despite giving an effective stimulating agent, confirm a diagnosis of AGHD.
AEZS-130 has been granted orphan-drug designation by the FDA for use as a diagnostic test for growth hormone deficiency. Aeterna Zentaris owns the worldwide rights to AEZS-130 which, if approved, would become the first orally administered diagnostic test for AGHD, the firm claims.