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Macrilen™ (macimorelin) 60 mg icon of pages representing data and information found during the clinical trial process

How was Macrilen™ studied?

Macrilen™ was studied in 140 adults in a head-to-head trial versus the current standard of care, the insulin tolerance test (ITT).1,2,a,b The positive agreement refers to the percentage of subjects who tested positive for AGHD with both tests, while the negative agreement correlates to the percentage of subjects who tested negative on both tests.

How was Macrilen™ studied?

Macrilen™ was studied in 140 adults in a head-to-head trial versus the current standard of care, the insulin tolerance test (ITT).1,2,a,b The positive agreement refers to the percentage of subjects who tested positive for AGHD with both tests, while the negative agreement correlates to the percentage of subjects who tested negative on both tests.

Macrilen™ (macimorelin) 60 mg icon of pages representing data and information found during the clinical trial process
  • Open label
  • Multicenter
  • Randomized
  • Crossover
  • Single dose

Co-primary endpoints

Percentage of positive and negative agreement between Macrilen™ and ITT based on prespecified GH cut-point values

Positive agreement, subjects with both a positive ITT and Macrilen™ test. Negative agreement, 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

  • Open label
  • Multicenter
  • Randomized
  • Crossover
  • Single dose

Co-primary endpoints

Percentage of positive and negative agreement between Macrilen™ and ITT based on prespecified GH cut-point values

Positive agreement, subjects with both a positive ITT and Macrilen™ test. Negative agreement, 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

The prespecified cut-point value for a diagnosis of AGHD with Macrilen™ was a maximally stimulated serum GH level of <2.8 ng/mL for the 4 blood draws.1

The prespecified cut-point value for a diagnosis of AGHD with Macrilen™ was a maximally stimulated serum GH level of <2.8 ng/mL for the 4 blood draws.1

aEnrolled safety and modified intention-to-treat (mITT) populations: Of 166 enrolled subjects, 157 underwent one stimulation test and formed the safety population. 154 subjects had both stimulation tests at least once. Of 157 subjects, 17 did not fulfill the mITT criterion (randomized subjects in whom both tests of the crossover were evaluable).2

bStudied in adult subjects with different pretest probabilities of GH deficiency (GHD) and in healthy control subjects.1

Who was tested for AGHD?

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

Who was tested for AGHD?

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

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
  • 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

Intermediate likelihood of GHD

(n=37)

  • Subjects who do not qualify for either high or low likelihood
  • Subjects who do not qualify for either high or low likelihood

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
  • 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

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

Overall diagnostic accuracy of Macrilen™

Correlation overall in those with a high likelihood of AGHD1

Table showing the diagnostic accuracy of Macrilen™ (macimorelin) 60 mg

Overall diagnostic accuracy of Macrilen™

Correlation overall 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%

Illustration showing the success rate of Macrilen™ (macimorelin) 60 mg testing
Macrilen™ (macimorelin) 60 mg illustration showing the success rate of insulin tolerance testing (ITT)

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

Macrilen™ results were reproducible in more than 91% of patients tested.1

Macrilen™ results were reproducible in more than 91% of patients tested.1

Well-tolerated safety profile in a clinical trial2

Common adverse events reported in <5% of subjects1

Well-tolerated safety profile in a clinical trial2

Common adverse events reported in <5% of subjects1

Table representing the most common adverse events (AEs) and percentages of patients (N=154) who experienced them
Table representing the most common adverse events (AEs) and percentages of patients (N=154) who experienced them

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

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

How to prepare for dosing

Macrilen™ (macimorelin) 60 mg icon indicating clinical testing and associated data

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

How to prepare for dosing

Macrilen™ (macimorelin) 60 mg icon indicating clinical testing and associated data

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

How to order

Macrilen™ (macimorelin) 60 mg icon of an order form page representing the ordering process

Learn how you can start ordering Macrilen™ for your patients.

How to order

Macrilen™ (macimorelin) 60 mg icon of an order form page representing the ordering process

Learn how you can start ordering Macrilen™ for your patients.

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 Prescribing Information

References

  1. Macrilen [prescribing information]. Plainsboro, NJ: Novo Nordisk Inc; 2018.
  2. Garcia JM, Biller BMK, Korbonits M, et al. Macimorelin as a diagnostic test for adult GH deficiency. J Clin Endocrinol Metab. 2018;103(8):3083-3093.