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FDA Rejects Growth Hormone Test
The FDA has given “thumbs down” to a new drug application (NDA) for macimorelin (Macrilen), an investigational, orally active ghrelin agonist, for use in evaluating adult growth hormone deficiency (AGHD). Based on its review, the FDA determined that the NDA could not be approved in its present form.
The agency’s complete response letter (CRL) mentioned that the planned analysis of the drug’s pivotal trial did not meet its stated primary efficacy objective. 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 these deficiencies, the CRL stated that the product’s developer (Aeterna Zentaris Inc.) will need to demonstrate the efficacy of macimorelin as a diagnostic test for AGHD in a new, confirmatory clinical study.
The CRL also noted that a serious event of electrocardiogram QT-interval prolongation occurred, for which attribution to the drug could not be excluded. Therefore, a dedicated QT study to evaluate the effect of macimorelin on the QT interval will also be necessary.
AGHD affects approximately 75,000 individuals in 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. The disorder is usually characterized by a reduction in bone mineral density, in lean body mass, in exercise capacity, and in overall quality of life.
Macimorelin is an orally active small molecule that stimulates the secretion of growth hormone. The product was granted “orphan drug” status by the FDA for AGHD.
Source: Aeterna Zentaris Inc.; November 6, 2014.