Aeterna Zentaris announced
they have received a Complete Response Letter from the U.S. Food and Drug Administration (FDA) regarding its New Drug Application (NDA) for MacrilenTM
(macimorelin), a novel orally-active ghrelin agonist that was intended to help in diagnosing/evaluating adult growth hormone deficiency (AGHD).
A Complete Response Letter means that the FDA has stopped reviewing the NDA for macimorelin.
The Complete Response Letter has not been made public at the time of writing but according to Aeterna Zentaris
, the FDA wrote:
“The planned analysis of the Company's pivotal trial did not meet its stated primary efficacy objective as agreed to in the Special Protocol Assessment agreement letter between the Company and the FDA. The Complete Response Letter further mentioned issues related to the lack of complete and verifiable source data for determining whether patients were accurately diagnosed with AGHD.”
In the Complete Response Letter, the FDA also wrote:
"In light of the failed primary analysis and data deficiencies noted, the clinical trial does not by itself support the indication."
The Complete Response Letter concluded that the Aeterna Zentaris will need to demonstrate the efficacy of macimorelin as a diagnostic test for growth hormone deficiency in a new, confirmatory clinical study.
David Dodd, Chairman and CEO at Aeterna Zentaris said:
"Following the FDA's decision, we are currently reviewing the outstanding issues stated in the Complete Reponse Letter in order to evaluate our options and future plans for MacrilenTM."
A conference call is planned for Friday November 7, 2014 at 9:00 am (EST) to discuss the company’s plans. For more details, visit www.aezsinc.com
in the "Newsroom" section.
In a press release
from 2012, the company indicated that their drug was ‘safe and effective in diagnosing AGHD.”
Adult Growth Hormone Deficiency (AGHD)
AGHD affects approximately 75,000 adults across the US, Canada and Europe. 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.