QUÉBEC CITY, Canada I November 6, 2014 I Aeterna Zentaris Inc. (NASDAQ: AEZS, TSX: AEZ) (the “Company”) 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 MacrilenTM (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 MacrilenTM.”
Conference call
Management will be hosting a conference call for the investment community beginning at 9:00 am (Eastern Time), on Friday, November 7, 2014, to discuss MacrilenTM‘s NDA. Individuals interested in participating in the live conference call by telephone may dial, in Canada, 514-807-9895 or 647-427-7450, outside Canada, 888-231-8191. They may also listen through the Internet at www.aezsinc.com in the “Newsroom” section. A replay will be available on the Company’s website for 30 days following the live event.
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 MacrilenTM (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.
SOURCE: Aeterna Zentaris
Post Views: 58
QUÉBEC CITY, Canada I November 6, 2014 I Aeterna Zentaris Inc. (NASDAQ: AEZS, TSX: AEZ) (the “Company”) 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 MacrilenTM (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 MacrilenTM.”
Conference call
Management will be hosting a conference call for the investment community beginning at 9:00 am (Eastern Time), on Friday, November 7, 2014, to discuss MacrilenTM‘s NDA. Individuals interested in participating in the live conference call by telephone may dial, in Canada, 514-807-9895 or 647-427-7450, outside Canada, 888-231-8191. They may also listen through the Internet at www.aezsinc.com in the “Newsroom” section. A replay will be available on the Company’s website for 30 days following the live event.
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 MacrilenTM (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.
SOURCE: Aeterna Zentaris
Post Views: 58
