Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZS) (the "Company") today announced that, following its meeting with the U.S. Food and Drug Administration (the "FDA" or the "Agency") on March 29, 2017, the Company intends to file a new drug application ("NDA") seeking approval of Macrilen™ (macimorelin) for the evaluation of growth hormone deficiency in adults ("AGHD").
Commenting on the meeting, Dr. Richard Sachse, the Company’s Chief Scientific Officer, stated, "During our meeting with the FDA, the Agency stated that the clinical studies we performed with respect to Macrilen™ address the prior deficiencies mentioned in the November 2014 complete response letter. This conclusion paves the way for our re-submission of an NDA for Macrilen™, which we expect to file in the third quarter of this year. While indicating that our conclusions regarding the performance of Macrilen™ are review issues subject to an examination of the complete data set, the Agency indicated that the summary data we submitted prior to the meeting appear to support the propositions we advanced. Most importantly, the FDA specified the additional statistical analysis of existing data that would be required to further support our conclusions. We expect that we can provide those data in a compelling fashion and demonstrate that Macrilen™ is a robust, repeatable test, demonstrating adequate sensitivity and specificity and that the performance of the product would be improved by utilizing a more appropriate cut-off point."
David A. Dodd, President and Chief Executive Officer of the Company stated, "We were encouraged by our dialogue with the FDA regarding Macrilen™. The Agency provided very helpful guidance to us that should help us in the registration process. Of course, the FDA will thoroughly review all data we provide with our NDA and make a decision regarding the approval of the product after doing so. Although there can be no assurance of approval of any NDA, we believe that we are now one important step closer to the commercialization of Macrilen™ in the U.S., providing a much needed new option and alternative to the ITT."
About the Study
The confirmatory Phase 3 clinical study of Macrilen™, entitled Confirmatory validation of oral macimorelin as a growth hormone (GH) stimulation test (ST) for the diagnosis of adult growth hormone deficiency (AGHD) in comparison with the insulin tolerance test (ITT), was designed as a two-way crossover study with the ITT as the benchmark comparator and involved some 26 sites in the U.S. and Europe. The trial involved 157 subjects, of whom 140 completed two evaluable tests for AGHD using both Macrilen™ and the ITT. Thirty-four of the patients were evaluated using Macrilen™ a second time to measure the repeatability of the result obtained using Macrilen™ as the evaluation method. The study population consisted of 115 patients who were suspected of having AGHD as a result of the presence of one or more symptoms or risk factors. This segment of the population included a range of patients from those considered at low risk of having AGHD to those considered at high risk. The study population also included 25 healthy subjects, who had no risk of having AGHD. Under the study protocol, the evaluation of AGHD with Macrilen™ will be considered successful, if the lower bound of the two-sided 95% confidence interval (or lower bound of the one-sided 97.5% confidence interval) for the primary efficacy variables is 75% or higher for "percent negative agreement", and 70% or higher for the "percent positive agreement". Based on meetings with the FDA as well as the European Medicines Agency ("EMA") and subsequent written scientific advice, the Company believes that the study meets the FDA’s and the EMA’s study-design expectations allowing U.S. and European approval if other conditions are met. Dr. Jose M. Garcia, MD, PhD, an Associate Professor of Medicine at the Puget Sound VA Hospital and the University of Washington in Seattle, was the principal investigator of the confirmatory Phase 3 clinical trial. More details about the trial are available at the following link: https://www.clinicaltrials.gov/ct2/show/NCT02558829?term=macimorelin&rank=1.
About Macrilen (macimorelin)
Macimorelin, a ghrelin agonist, is an orally-active small molecule that stimulates the secretion of growt...
(All Rights Reserved)