Aeterna Zentaris Inc., a specialty biopharmaceutical company, is planning to conduct a new, confirmatory phase 3 clinical study to demonstrate the efficacy of Macrilen (macimorelin), a novel orally-active ghrelin agonist for use in evaluating adult growth hormone deficiency (AGHD), as well as a dedicated thorough QT study to evaluate the effect of Macrilen on myocardial repolarisation.
The decision follows a positive and helpful meeting with the U.S. Food and Drug Administration (FDA) regarding its New Drug Application for Macrilen. The company call a panel of US and EU endocrinology experts. The panel advised the company to continue to seek approval for the compound because of their confidence in its efficacy and because there currently is no FDA-approved diagnostic test for AGHD.
After the meeting, company and the FDA agreed on the general design of the confirmatory study as well as evaluation criteria. The study will be conducted as a two-way crossover with the insulin tolerance test as the benchmark comparator. The study population will consist of patients with a medical history documenting risk factors for AGHD and will include a spectrum of patients from those with a low risk of having AGHD to those with a high risk of having the condition. The company will submit a proposed final protocol to the FDA for approval prior to commencing the confirmatory study.
The completion of the confirmatory Phase 3 study and the QT study will take about 18 months and will require a combined expenditure of between $5 million and $6 million. The company's goal is to conduct a phase 3 study that will satisfy the registration requirements of the European Medicines Agency (EMA) as well as the FDA. The company expects to receive comments from the EMA regarding the study design during a scientific advice meeting in early May.
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