Macrilen™ (macimorelin) for oral solution was studied in 140 adults in a head-to-head trial vs. the insulin tolerance test (ITT).^1,a,b

^aThe diagnostic efficacy of the Macrilen™ test was established in a randomized, open-label, single-dose, cross-over study. The objective of the study was to compare the level of agreement between Macrilen™ test results and ITT results in adult patients with different pre-test probability of growth hormone deficiency and healthy control subjects. Each participant was to undergo the two diagnostic tests and serve as his or her own control. One hundred and fifty-seven subjects underwent at least one of the two tests in this study. Data on both tests were available for 140 subjects. One out of 154 Macrilen™ tests performed failed due to a technical error and 27 out of 157 ITTs performed failed because induction of severe hypoglycemia could not be achieved.¹
^bStudied in adult subjects with different pretest probabilities of GH deficiency (GHD) and in healthy control subjects.¹

In the head-to-head trial versus ITT, adults were segmented into the following groups to compare levels of agreement between Macrilen™ test results and ITT results.¹

Group A

High likelihood of GHD (n=38)

Structural hypothalamic or pituitary lesions and low insulin-like growth factor-1 (IGF-1)
3 or more pituitary hormone deficiencies and low IGF-1
Childhood-onset GHD with structural lesions and low IGF-1

Group B

Intermediate likelihood of GHD (n=37)

Subjects who do not qualify for either high or low likelihood

Group C

Low likelihood of GHD (n=40)

Subjects with only 1 risk factor for GHD, such as
- History of distant traumatic brain injury
- Only 1 pituitary hormone deficiency with otherwise normal pituitary function
Isolated idiopathic childhood-onset GHD without additional pituitary deficits

Group D

Healthy controls (n=25)

Healthy subjects matching subjects in high-likelihood group by sex, age (±5 years), BMI (±2 kg/m²), and estrogen status (females only)

Correlation overall and in those with a high likelihood of AGHD¹

Type of correlation

All subjects

High likelihood of AGHD

Positive

74%

89%

Negative

94%

100%

Overall

84%

89%

Positive correlation, subjects with both a positive ITT and Macrilen™ test. Negative correlation, subjects with both a negative ITT and Macrilen™ test. All subjects included those with a high (n=38), intermediate (n=37), or low (n=40) likelihood of AGHD, as well as healthy controls (n=25).¹

^cDefined as neuroglycopenic signs confirmed by glucose less than 40 mg/dL.

Macrilen™ reproducible in approximately 91% of patients tested (n=34).¹

Common adverse events (AEs) reported in the Phase III clinical trial in <5% of subjects¹

Adverse events

Number of subjects (n=154)

Percentage of subjects

Dysgeusia

7

4.5%

Dizziness

6

3.9%

Headache

6

3.9%

Fatigue

6

3.9%

Nausea

5

3.2%

Hunger

5

3.2%

Diarrhea

3

1.9%

Upper respiratory tract infection

3

1.9%

Feeling hot

2

1.3%

Hyperhidrosis

2

1.3%

Nasopharyngitis

2

1.3%

Sinus bradycardia

2

1.3%

No AEs led to failure to complete the Macrilen™ test.¹

Review the step-by-step process for preparing and administering Macrilen™.

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Selected Important Safety Information for Macrilen™

Warnings and Precautions

QT Prolongation: Macrilen™ causes an increase of about 11 msec in the corrected QT (QTc) interval. QT prolongation can lead to development of torsade de pointes-type ventricular tachycardia with the risk increasing as the degree of prolongation increases. The concomitant use of Macrilen™ with drugs that are known to prolong the QT interval should be avoided

Indication and Limitation of Use

Macrilen™ (macimorelin) 60 mg for oral solution is indicated for the diagnosis of adult growth hormone deficiency (AGHD).

The safety and diagnostic performance of Macrilen™ have not been established for subjects with a body mass index (BMI) >40 kg/m²

Important Safety Information

Warnings and Precautions

QT Prolongation: Macrilen™ causes an increase of about 11 msec in the corrected QT (QTc) interval. QT prolongation can lead to development of torsade de pointes-type ventricular tachycardia with the risk increasing as the degree of prolongation increases. The concomitant use of Macrilen™ with drugs that are known to prolong the QT interval should be avoided
Potential for False Positive Test Results with Use of Strong CYP3A4 Inducers: Concomitant use of strong CYP3A4 inducers with Macrilen™ can decrease macimorelin plasma levels significantly and thereby lead to a false positive result. Strong CYP3A4 inducers should be discontinued and enough time should be given to allow washout of CYP3A4 inducers prior to test administration
Potential for False Negative Test Results in Recent Onset Hypothalamic Disease: Adult growth hormone (GH) deficiency caused by a hypothalamic lesion may not be detected early in the disease process. Macimorelin acts downstream from the hypothalamus and macimorelin stimulated release of stored GH reserves from the anterior pituitary could produce a false negative result early when the lesion involves the hypothalamus. Repeat testing may be warranted in this situation

Adverse Reactions

The most common adverse reactions are dysgeusia, dizziness, headache, fatigue, nausea, hunger, diarrhea, upper respiratory tract infection, feeling hot, hyperhidrosis, nasopharyngitis, and sinus bradycardia

Please click here for Macrilen™ Prescribing Information.

References:

Macrilen [prescribing information]. Plainsboro, NJ: Novo Nordisk Inc; 2021.

How was Macrilen™ studied?

How was Macrilen™ studied?

How was accuracy measured?

Who was tested for adult growth hormone deficiency (AGHD)?

Overall diagnostic accuracy of Macrilen™

Demonstrated safety profile in a clinical trial1

How to prepare for dosing

How to order

Selected Important Safety Information for Macrilen™

Warnings and Precautions

Indication and Limitation of Use

Important Safety Information

Warnings and Precautions

Adverse Reactions

Demonstrated safety profile in a clinical trial¹