Evidence memo / GH / IGF axis

Macimorelin

Also: Macrilen · Ghryvelin · AEZS-130 · macimorelin acetate · ghrelin receptor agonist

Macimorelin (Macrilen) is an oral, ghrelin-like drug used as a one-time test, not a treatment. Swallowed as a single dose, it triggers a burst of growth hormone in a healthy pituitary, so doctors can tell whether an adult is genuinely growth-hormone deficient. The Watchlist boundary is that it is a diagnostic given once under supervision, and the telling contrast to the ghrelin-receptor peptides (MK-677, ipamorelin) sold for chronic growth-hormone boosting: same receptor, but the approved use is a single diagnostic dose.

Approved for specific usesApproved specific use

The verdict

Our read at a glance.

Whether macimorelin is understood as an approved oral ghrelin-receptor agonist used as a one-time growth-hormone stimulation test for adult growth-hormone deficiency, rather than a growth-hormone-boosting or anti-aging treatment.

The signal
A head-to-head validation trial compared the oral macimorelin test against the insulin tolerance test, the long-standing reference for diagnosing adult growth-hormone deficiency, and found good agreement with high sensitivity and specificity and reproducible results, in a much simpler and safer oral protocol. That evidence is what earned its approval.
The unknown
The open questions are practical: growth-hormone responses are blunted in people with obesity, which complicates any stimulation test and its cutoff; strong drug interactions can cause false results; and performance in borderline or partial deficiency is less clean. Its role as a diagnostic is settled, and there is no role as a treatment.
Our read
The rare oral ghrelin-receptor agonist that actually won approval, but only as a one-time test, not a treatment. Macimorelin reliably provokes a growth-hormone surge to show whether the pituitary can still make growth hormone, giving a safer oral alternative to the insulin tolerance test. Read it as a diagnostic, and as the telling contrast to the ghrelin-receptor peptides (MK-677, ipamorelin) sold for chronic boosting: same receptor, but the approved use is a single diagnostic dose.

The mechanism

How it works.

The body's own hunger hormone, ghrelin, also tells the pituitary to release growth hormone by switching on a receptor on the gland's growth-hormone-making cells. Macimorelin is a small, orally active molecule built to act like ghrelin at that receptor. Swallowed as a single weight-based dose after fasting, it triggers a burst of growth hormone in someone whose pituitary is healthy; in adult growth-hormone deficiency the gland cannot mount that surge, so the blood growth-hormone level stays low. By measuring growth hormone in the blood over the 90 minutes after the drink, clinicians can tell a normal response from a deficient one. Because it works through the same ghrelin receptor that the popular growth-hormone-secretagogue peptides target, it is chemically in their family, but it is used once, to measure, not repeatedly to boost.

Safety & regulatory boundary

The consequential part.

Macimorelin modestly prolongs the heart's QT interval, so other QT-prolonging drugs are avoided around the test, and strong CYP3A4 inducers lower its levels and can cause a falsely low (false-positive) growth-hormone result, so they are stopped beforehand. It is an oral diagnostic agent given as a single dose under medical supervision, not a medicine taken over time. Macrilen is not the same as research-labeled macimorelin or the unapproved ghrelin-receptor peptides (MK-677, ipamorelin, GHRP) marketed for muscle or anti-aging, and its approval covers a one-time test only, not growth-hormone or IGF-1 enhancement.

What's next

What we're watching.

Real-world accuracy across body-weight ranges and in partial deficiency, drug-interaction false-positive reporting, and whether the oral test fully replaces the insulin tolerance test in practice.

Context

Why this is discussed.

People in the peptide world chase oral ghrelin-receptor agonists like MK-677 to push up growth hormone. Macimorelin is the one oral ghrelin-receptor agonist that actually earned FDA approval, but tellingly only as a diagnostic test used a single time, which is a clear-eyed contrast to how the same receptor is marketed for muscle and anti-aging.

The evidence base

19 cited references.

Regulatory & labels2

DailyMed label: Macrilen

Novo Nordisk (FDA label) · 2021

DailyMed label: Macrilen

Aeterna Zentaris GmbH (FDA label) · 2026

Indexed literature (PubMed)11

Macimorelin as a Diagnostic Test for Adult GH Deficiency

Garcia JM et al. · J Clin Endocrinol Metab · 2018

An update on the diagnosis of growth hormone deficiency

Gabreanu GR · Discoveries (Craiova) · 2018

Growth hormone deficiency in adults with Cushing's disease

Tritos NA · Best Pract Res Clin Endocrinol Metab · 2021

Keep reading

Related published memos.