MacrilenTreatment for Diagnosis of Adult Growth Hormone Deficiency
FDA Issues Complete Response Letter for Macrilen (macimorelin) NDA in Adult Growth Hormone Deficiency
Québec City, Canada, November 6, 2014 – Aeterna Zentaris Inc. today announced that the Company has received a Complete Response Letter (“CRL”) from the U.S. Food and Drug Administration (“FDA”) for its New Drug Application (“NDA”) for Macrilen (macimorelin), a novel orally-active ghrelin agonist, for use in evaluating adult growth hormone deficiency (“AGHD”). Based on its review, the FDA has determined that the NDA cannot be approved in its present form.
The CRL mentions that 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 CRL further mentioned issues related to the lack of complete and verifiable source data for determining whether patients were accurately diagnosed with AGHD. The FDA concluded that, “in light of the failed primary analysis and data deficiencies noted, the clinical trial does not by itself support the indication.” To address the deficiencies identified above, the CRL states that the Company will need to demonstrate the efficacy of macimorelin as a diagnostic test for growth hormone deficiency in a new, confirmatory clinical study.
The CRL also outlined that a serious event of electrocardiogram QT interval prolongation occurred for which attribution to drug could not be excluded. Therefore a dedicated thorough QT study to evaluate the effect of macimorelin on the QT interval would be necessary.
David Dodd, Chairman and CEO at Aeterna Zentaris said, “Following the FDA's decision, we are currently reviewing the outstanding issues stated in the CRL in order to evaluate our options and future plans for Macrilen.”
About Adult Growth Hormone Deficiency (AGHD)
AGHD affects approximately 75,000 adults across the U.S., Canada and Europe. Growth hormone 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.
About Macrilen (macimorelin)
Macimorelin, a ghrelin agonist, is a novel orally-active small molecule that stimulates the secretion of growth hormone. Macimorelin, under the trade name MacrilenTM, has been granted orphan drug designation by the FDA for AGHD. Aeterna Zentaris owns the worldwide rights to this novel patented compound.
About Aeterna Zentaris
Aeterna Zentaris is a specialty biopharmaceutical company engaged in developing and commercializing novel treatments in oncology and endocrinology. For more information, visit www.aezsinc.com.
This press release contains forward-looking statements made pursuant to the safe harbor provisions of the US Securities Litigation Reform Act of 1995. Forward-looking statements involve known and unknown risks and uncertainties that could cause the Company's actual results to differ materially from those in the forward-looking statements. Such risks and uncertainties include, among others, the availability of funds and resources to pursue R&D projects, the successful and timely completion of clinical studies, the risk that safety and efficacy data from any of our Phase 3 trials may not coincide with the data analyses from previously reported Phase 1 and/or Phase 2 clinical trials, the ability of the Company to efficiently commercialize one or more of its products or product candidates, the ability of the Company to take advantage of business opportunities in the pharmaceutical industry, uncertainties related to the regulatory process, the specific outcome of our recently announced global resources optimization program and the financial impact on the Company resulting therefrom, and general changes in economic conditions. Investors should consult the Company's quarterly and annual filings with the Canadian and US securities commissions for additional information on risks and uncertainties relating to forward-looking statements. Investors are cautioned not to rely on these forward-looking statements. The Company does not undertake to update these forward-looking statements. We disclaim any obligation to update any such factors or to publicly announce the result of any revisions to any of the forward-looking statements contained herein to reflect future results, events or developments, unless required to do so by a governmental authority or by applicable law.
Source: Aeterna Zentaris Inc.
Posted: November 2014
- Aeterna Zentaris Announces FDA Approval of Macrilen (macimorelin) for Diagnosis of Adult Growth Hormone Deficiency - December 20, 2017
- NDA for Macrilen for the Evaluation of Growth Hormone Deficiency in Adults Granted December 30, 2017 PDUFA Date - July 18, 2017
- Aeterna Zentaris Resubmits NDA for Macrilen for the Evaluation of Growth Hormone Deficiency in Adults - June 30, 2017
- Aeterna Zentaris Intends to File NDA with Respect to Macrilen in Third Quarter of 2017 - March 30, 2017
- Aeterna Zentaris Announces Plans to Pursue FDA Registration of Macrilen - February 13, 2017
- Aeterna Zentaris Completes Patient Recruitment for Confirmatory Phase 3 Trial of Macrilen - October 26, 2016
- Aeterna Zentaris Announces Plans to Initiate Phase 3 Study of Macrilen in AGHD - April 13, 2015
- Aeterna Zentaris NDA for Macimorelin Acetate in AGHD Accepted for Filing by the FDA - January 6, 2014
- Aeterna Zentaris Submits New Drug Application to FDA for Macimorelin Acetate (AEZS-130) for Evaluation of AGHD - November 5, 2013