Macimorelin for Diagnosis of Adult Growth Hormone Deficiency in EU


November 19, 2018

The Committee for Medicinal Products for Human Use of the European Medicines Agency (EMA) has issued a positive opinion on an orally available ghrelin agonist macimorelin (Macimorelin, Aeterna Zentaris) to be used in the diagnosis of patients with adult growth hormone deficiency.

Adult growth hormone deficiency is a rare disorder characterized by the inadequate secretion of growth hormone from the pituitary gland. It can be hereditary; acquired as a result of trauma, infection, radiation therapy, or brain tumor growth; or can even emerge without a diagnosable cause. Currently, it is treated with once-daily injections of subcutaneous growth hormone.

Considering a population of 510 million for the European Union, "research based on incidence prevalence suggests that at least 35,000 adults could be afflicted with GHD," says Aeterna Zentaris in a press release.

Macimorelin stimulates the secretion of growth hormone by the pituitary gland into the circulatory system. Stimulated growth hormone levels are measured in four blood samples over 90 minutes after oral administration of the agent for the assessment of growth hormone deficiency.

Macimorelin was approved for the same indication, diagnosis of adult growth hormone deficiency, in the United States in December 2017, where it is known under the brand name Macrilen.

Prior to the approval of macimorelin, the historical gold standard for evaluation of adult growth hormone deficiency was the insulin tolerance test, an intravenous test requiring many blood draws over several hours.

One of the benefits of macimorelin is its ability to confirm diagnosis of growth hormone deficiency with high sensitivity and specificity in adult patients, says the EMA.

Macimorelin is a peptide mimetic and will be available as 60-mg granules for oral solution.

The most common side effects are dysgeusia, dizziness, headache, fatigue, feeling hot, nausea, and diarrhea.

It is proposed that the use of macimorelin be supervised by a physician or healthcare professional experienced in diagnosing growth hormone deficiency, says EMA.

Detailed recommendations for the use of this product will be described in the summary of product characteristics, which will be published in the European public assessment report and made available in all official European Union languages after marketing authorization has been granted by the European Commission.

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