U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS

Details

Stereochemistry ABSOLUTE
Molecular Formula C26H30N6O3
Molecular Weight 474.5558
Optical Activity UNSPECIFIED
Defined Stereocenters 2 / 2
E/Z Centers 0
Charge 0

SHOW SMILES / InChI
Structure of MACIMORELIN

SMILES

CC(C)(C(=N[C@]([H])(Cc1c[nH]c2ccccc12)C(=N[C@]([H])(Cc3c[nH]c4ccccc34)N=CO)O)O)N

InChI

InChIKey=UJVDJAPJQWZRFR-DHIUTWEWSA-N
InChI=1S/C26H30N6O3/c1-26(2,27)25(35)31-22(11-16-13-28-20-9-5-3-7-18(16)20)24(34)32-23(30-15-33)12-17-14-29-21-10-6-4-8-19(17)21/h3-10,13-15,22-23,28-29H,11-12,27H2,1-2H3,(H,30,33)(H,31,35)(H,32,34)/t22-,23-/m1/s1

HIDE SMILES / InChI
Macimorelin (AEZS 130) is an orally active, small-molecule, peptidomimetic growth hormone secretagogue receptor (GHSR1A) agonist (ghrelin analogue), being developed by AEterna Zentaris for the diagnosis of adult growth hormone deficiency (AGHD; somatotropin deficiency), and for the treatment of cachexia associated with chronic disease such as AIDS and cancer. Macimorelin was approved by the FDA in December 2017 under the market name Macrilen for oral solution. Macimorelin stimulates GH release by activating growth hormone secretagogue receptors present in the pituitary and hypothalamus. Macimorelin has been granted orphan drug designation by the FDA for diagnosis of AGHD.

Approval Year

TargetsConditions

Conditions

ConditionModalityTargetsHighest PhaseProduct
Diagnostic
MACRILEN

Approved Use

MACRILEN is a growth hormone (GH) secretagogue receptor agonist indicated for the diagnosis of adult growth hormone deficiency

Launch Date

1.51364161E12
Cmax

Cmax

ValueDoseCo-administeredAnalytePopulation
9.24 ng/mL
0.5 mg single, oral
dose: 0.5 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
[NO STEREO] MACIMORELIN plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: FEMALE / MALE
food status: FASTED
10.6 μg/L
0.5 mg/kg 1 times / day multiple, oral
dose: 0.5 mg/kg
route of administration: Oral
experiment type: MULTIPLE
co-administered:
[NO STEREO] MACIMORELIN plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: FEMALE / MALE
food status: FASTED
9.17 ng/mL
0.5 mg/kg single, oral
dose: 0.5 mg/kg
route of administration: Oral
experiment type: SINGLE
co-administered:
MACIMORELIN plasma
Homo sapiens
population: UNHEALTHY
age: ADULT
sex: FEMALE / MALE
food status: FASTED
21.9 ng/mL
2 mg/kg single, oral
dose: 2 mg/kg
route of administration: Oral
experiment type: SINGLE
co-administered:
MACIMORELIN plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: FEMALE / MALE
food status: FASTED
AUC

AUC

ValueDoseCo-administeredAnalytePopulation
38 ng × h/mL
0.5 mg single, oral
dose: 0.5 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
[NO STEREO] MACIMORELIN plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: FEMALE / MALE
food status: FASTED
32 μg × h/L
0.5 mg/kg 1 times / day multiple, oral
dose: 0.5 mg/kg
route of administration: Oral
experiment type: MULTIPLE
co-administered:
[NO STEREO] MACIMORELIN plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: FEMALE / MALE
food status: FASTED
59.45 ng × h/mL
0.5 mg/kg single, oral
dose: 0.5 mg/kg
route of administration: Oral
experiment type: SINGLE
co-administered:
MACIMORELIN plasma
Homo sapiens
population: UNHEALTHY
age: ADULT
sex: FEMALE / MALE
food status: FASTED
75.7 ng × h/mL
2 mg/kg single, oral
dose: 2 mg/kg
route of administration: Oral
experiment type: SINGLE
co-administered:
MACIMORELIN plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: FEMALE / MALE
food status: FASTED
T1/2

T1/2

ValueDoseCo-administeredAnalytePopulation
3.51 h
0.5 mg single, oral
dose: 0.5 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
[NO STEREO] MACIMORELIN plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: FEMALE / MALE
food status: FASTED
3.9 h
0.5 mg/kg 1 times / day multiple, oral
dose: 0.5 mg/kg
route of administration: Oral
experiment type: MULTIPLE
co-administered:
[NO STEREO] MACIMORELIN plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: FEMALE / MALE
food status: FASTED
3.76 h
0.5 mg/kg single, oral
dose: 0.5 mg/kg
route of administration: Oral
experiment type: SINGLE
co-administered:
MACIMORELIN plasma
Homo sapiens
population: UNHEALTHY
age: ADULT
sex: FEMALE / MALE
food status: FASTED
7.23 h
2 mg/kg single, oral
dose: 2 mg/kg
route of administration: Oral
experiment type: SINGLE
co-administered:
MACIMORELIN plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: FEMALE / MALE
food status: FASTED
Funbound

Funbound

ValueDoseCo-administeredAnalytePopulation
22%
0.5 mg/kg 1 times / day multiple, oral
dose: 0.5 mg/kg
route of administration: Oral
experiment type: MULTIPLE
co-administered:
[NO STEREO] MACIMORELIN plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: FEMALE / MALE
food status: FASTED
Doses

Doses

DosePopulationAdverse events​
2 mg/kg 1 times / day single, oral
Highest studied dose
Dose: 2 mg/kg, 1 times / day
Route: oral
Route: single
Dose: 2 mg/kg, 1 times / day
Sources: Page: 205598Orig1s000MedR.pdf - p.124
healthy, adult
n = 9
Health Status: healthy
Age Group: adult
Population Size: 9
Sources: Page: 205598Orig1s000MedR.pdf - p.124
Other AEs: Diarrhoea, Pain...
Other AEs:
Diarrhoea (11.1%)
Pain (11.1%)
Headache (44.4%)
Sources: Page: 205598Orig1s000MedR.pdf - p.124
0.5 mg/kg 1 times / day single, oral
Recommended
Dose: 0.5 mg/kg, 1 times / day
Route: oral
Route: single
Dose: 0.5 mg/kg, 1 times / day
Sources: Page: 205598Orig1s000MedR.pdf - p.124, p.52
unhealthy, adult
n = 52
Health Status: unhealthy
Condition: adult growth hormone deficiency
Age Group: adult
Population Size: 52
Sources: Page: 205598Orig1s000MedR.pdf - p.124, p.52
Disc. AE: Vein collapse...
AEs leading to
discontinuation/dose reduction:
Vein collapse (1.9%)
Sources: Page: 205598Orig1s000MedR.pdf - p.124, p.52
AEs

AEs

AESignificanceDosePopulation
Diarrhoea 11.1%
2 mg/kg 1 times / day single, oral
Highest studied dose
Dose: 2 mg/kg, 1 times / day
Route: oral
Route: single
Dose: 2 mg/kg, 1 times / day
Sources: Page: 205598Orig1s000MedR.pdf - p.124
healthy, adult
n = 9
Health Status: healthy
Age Group: adult
Population Size: 9
Sources: Page: 205598Orig1s000MedR.pdf - p.124
Pain 11.1%
2 mg/kg 1 times / day single, oral
Highest studied dose
Dose: 2 mg/kg, 1 times / day
Route: oral
Route: single
Dose: 2 mg/kg, 1 times / day
Sources: Page: 205598Orig1s000MedR.pdf - p.124
healthy, adult
n = 9
Health Status: healthy
Age Group: adult
Population Size: 9
Sources: Page: 205598Orig1s000MedR.pdf - p.124
Headache 44.4%
2 mg/kg 1 times / day single, oral
Highest studied dose
Dose: 2 mg/kg, 1 times / day
Route: oral
Route: single
Dose: 2 mg/kg, 1 times / day
Sources: Page: 205598Orig1s000MedR.pdf - p.124
healthy, adult
n = 9
Health Status: healthy
Age Group: adult
Population Size: 9
Sources: Page: 205598Orig1s000MedR.pdf - p.124
Vein collapse 1.9%
Disc. AE
0.5 mg/kg 1 times / day single, oral
Recommended
Dose: 0.5 mg/kg, 1 times / day
Route: oral
Route: single
Dose: 0.5 mg/kg, 1 times / day
Sources: Page: 205598Orig1s000MedR.pdf - p.124, p.52
unhealthy, adult
n = 52
Health Status: unhealthy
Condition: adult growth hormone deficiency
Age Group: adult
Population Size: 52
Sources: Page: 205598Orig1s000MedR.pdf - p.124, p.52
OverviewDrug as perpetrator​

Drug as perpetrator​

TargetModalityActivityMetaboliteClinical evidence
no [IC50 25 uM]
no
no
no
no
no
no
no
yes
yes
yes
Drug as victimTox targets

Tox targets

TargetModalityActivityMetaboliteClinical evidence
PubMed

PubMed

TitleDatePubMed
The macimorelin-stimulated growth hormone test for adult growth hormone deficiency diagnosis.
2014 Jul
Patents

Sample Use Guides

Recommended dose is 0.5 mg/kg as a single oral dose, after fasting for at least 8 hours
Route of Administration: Oral
Macimorelin exhibited a high binding affinity to the cloned hGHS-R 1a receptor (IC50 value of 123 nM).
Name Type Language
MACIMORELIN
DASH   INN   USAN   WHO-DD  
INN   USAN  
Official Name English
EP-1572
Code English
MACIMORELIN [MI]
Common Name English
D-87575
Code English
JMV-1843
Code English
ARD-07
Code English
ARD 07
Code English
N(SUP 2)-(2-AMINO-2-METHYLPROPANOYL-N1-((1R)-1-FORMAMIDO-2-(1H-INDOL-3-YL)ETHYL)-D-TRYPTOPHANAMIDE
Common Name English
MACIMORELIN [USAN]
Common Name English
AEZS-130
Code English
MACIMORELIN [WHO-DD]
Common Name English
EP-01572
Code English
MACIMORELIN [INN]
Common Name English
EP 1572
Code English
Classification Tree Code System Code
WHO-ATC V04CD06
Created by admin on Sat Jun 26 02:25:29 UTC 2021 , Edited by admin on Sat Jun 26 02:25:29 UTC 2021
NCI_THESAURUS C76358
Created by admin on Sat Jun 26 02:25:29 UTC 2021 , Edited by admin on Sat Jun 26 02:25:29 UTC 2021
NDF-RT N0000193785
Created by admin on Sat Jun 26 02:25:29 UTC 2021 , Edited by admin on Sat Jun 26 02:25:29 UTC 2021
Code System Code Type Description
PUBCHEM
9804938
Created by admin on Sat Jun 26 02:25:29 UTC 2021 , Edited by admin on Sat Jun 26 02:25:29 UTC 2021
PRIMARY
NCI_THESAURUS
C95123
Created by admin on Sat Jun 26 02:25:29 UTC 2021 , Edited by admin on Sat Jun 26 02:25:29 UTC 2021
PRIMARY
RXCUI
1999420
Created by admin on Sat Jun 26 02:25:29 UTC 2021 , Edited by admin on Sat Jun 26 02:25:29 UTC 2021
PRIMARY
FDA UNII
8680B21W73
Created by admin on Sat Jun 26 02:25:29 UTC 2021 , Edited by admin on Sat Jun 26 02:25:29 UTC 2021
PRIMARY
DRUG BANK
DB13074
Created by admin on Sat Jun 26 02:25:29 UTC 2021 , Edited by admin on Sat Jun 26 02:25:29 UTC 2021
PRIMARY
WIKIPEDIA
MACIMORELIN
Created by admin on Sat Jun 26 02:25:29 UTC 2021 , Edited by admin on Sat Jun 26 02:25:29 UTC 2021
PRIMARY
CAS
381231-18-1
Created by admin on Sat Jun 26 02:25:29 UTC 2021 , Edited by admin on Sat Jun 26 02:25:29 UTC 2021
PRIMARY
DRUG CENTRAL
5271
Created by admin on Sat Jun 26 02:25:29 UTC 2021 , Edited by admin on Sat Jun 26 02:25:29 UTC 2021
PRIMARY
MERCK INDEX
M12057
Created by admin on Sat Jun 26 02:25:29 UTC 2021 , Edited by admin on Sat Jun 26 02:25:29 UTC 2021
PRIMARY
INN
9073
Created by admin on Sat Jun 26 02:25:29 UTC 2021 , Edited by admin on Sat Jun 26 02:25:29 UTC 2021
PRIMARY
ChEMBL
CHEMBL278623
Created by admin on Sat Jun 26 02:25:29 UTC 2021 , Edited by admin on Sat Jun 26 02:25:29 UTC 2021
PRIMARY