FDA CRL rejects Macrilen for Growth Hormone Deficiency-Aeterna Zentaris

Aeterna Zentaris has received a Complete Response Letter from the FDA for its New Drug Application for Macrilen (macimorelin), for use in evaluating adult Growth Hormone Deficiency. 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 company is now reviewing the outstanding issues stated in the CRL in order to evaluate options and future plans for Macrilen.