MACIMORELIN ACETATE

Details

Stereochemistry	ABSOLUTE
Molecular Formula	C26H30N6O3.C2H4O2
Molecular Weight	534.6068
Optical Activity	UNSPECIFIED
Defined Stereocenters	2 / 2
E/Z Centers	0
Charge	0

SHOW SMILES / InChI

Structure Search

SMILES

CC(O)=O.CC(C)(N)C(=O)N[C@H](CC1=CNC2=C1C=CC=C2)C(=O)N[C@H](CC3=CNC4=C3C=CC=C4)NC=O

InChI

InChIKey=WVDSKQXKCDZXLH-OHIDFYLOSA-N

InChI=1S/C26H30N6O3.C2H4O2/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;1-2(3)4/h3-10,13-15,22-23,28-29H,11-12,27H2,1-2H3,(H,30,33)(H,31,35)(H,32,34);1H3,(H,3,4)/t22-,23-;/m1./s1

HIDE SMILES / InChI

Molecular Formula	C26H30N6O3
Molecular Weight	474.5548
Charge	0
Count

Stereochemistry	ABSOLUTE
Additional Stereochemistry	No
Defined Stereocenters	2 / 2
E/Z Centers	0
Optical Activity	UNSPECIFIED

Molecular Formula	C2H4O2
Molecular Weight	60.052
Charge	0
Count

Stereochemistry	ACHIRAL
Additional Stereochemistry	No
Defined Stereocenters	0 / 0
E/Z Centers	0
Optical Activity	NONE

Description
Sources: https://www.accessdata.fda.gov/drugsatfda_docs/label/2017/205598s000lbl.pdf | https://www.drugbank.ca/drugs/DB13074

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.

CNS Activity

Originator

Approval Year

Targets

Primary Target	Pharmacology	Condition	Potency
Ghrelin receptor 15 Target ID: CHEMBL4616 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/label/2017/205598s000lbl.pdf \| https://www.drugbank.ca/drugs/DB13074 \| https://www.ncbi.nlm.nih.gov/pubmed/23559086	Agonist 2752		123.0 nM [IC50]

Conditions

Condition	Modality	Targets	Highest Phase	Product
Growth hormone deficiency 5 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/label/2017/205598s000lbl.pdf	Diagnostic		Approved 8583	MACRILEN Approved Use MACRILEN is a growth hormone (GH) secretagogue receptor agonist indicated for the diagnosis of adult growth hormone deficiency Launch Date 2017

C_max

Value	Dose	Analyte	Population
9.24 ng/mL REVIEW https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000ClinPharmR.pdf	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
21.9 ng/mL REVIEW https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000ClinPharmR.pdf	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
9.17 ng/mL REVIEW https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000ClinPharmR.pdf	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
10.6 μg/L REVIEW https://www.ema.europa.eu/en/documents/assessment-report/macimorelin-aeterna-zentaris-epar-public-assessment-report_en.pdf	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

AUC

Value	Dose	Analyte	Population
38 ng × h/mL REVIEW https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000ClinPharmR.pdf	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
75.7 ng × h/mL REVIEW https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000ClinPharmR.pdf	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
59.45 ng × h/mL REVIEW https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000ClinPharmR.pdf	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
32 μg × h/L REVIEW https://www.ema.europa.eu/en/documents/assessment-report/macimorelin-aeterna-zentaris-epar-public-assessment-report_en.pdf	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

T_1/2

Value	Dose	Analyte	Population
3.51 h REVIEW https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000ClinPharmR.pdf	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
7.23 h REVIEW https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000ClinPharmR.pdf	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
3.76 h REVIEW https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000ClinPharmR.pdf	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
3.9 h REVIEW https://www.ema.europa.eu/en/documents/assessment-report/macimorelin-aeterna-zentaris-epar-public-assessment-report_en.pdf	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

F_unbound

Value	Dose	Co-administered	Analyte	Population
22% REVIEW https://www.ema.europa.eu/en/documents/assessment-report/macimorelin-aeterna-zentaris-epar-public-assessment-report_en.pdf	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

Dose	Population	Adverse 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: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000MedR.pdf	healthy, adult Health Status: healthy Age Group: adult Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000MedR.pdf	Other AEs: Diarrhoea, Pain... Other AEs: Diarrhoea (11.1%) Pain (11.1%) Headache (44.4%) Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000MedR.pdf
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: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000MedR.pdf	unhealthy, adult Health Status: unhealthy Age Group: adult Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000MedR.pdf	Disc. AE: Vein collapse... AEs leading to discontinuation/dose reduction: Vein collapse (1.9%) Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000MedR.pdf

AEs

AE	Significance	Dose	Population
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: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000MedR.pdf	healthy, adult Health Status: healthy Age Group: adult Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000MedR.pdf
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: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000MedR.pdf	healthy, adult Health Status: healthy Age Group: adult Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000MedR.pdf
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: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000MedR.pdf	healthy, adult Health Status: healthy Age Group: adult Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000MedR.pdf
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: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000MedR.pdf	unhealthy, adult Health Status: unhealthy Age Group: adult Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000MedR.pdf

Overview

CYP3A4	CYP2C9	CYP2D6	hERG
substrate 1946 inhibitor 2252 inducer 1087	inhibitor 2438 substrate 1193	inhibitor 2507 substrate 1388	inhibitor 2217

OverviewOther

Other Inhibitor	Other Substrate	Other Inducer
CYP1A2 2153 CYP2A6 879 CYP2C8 1057 CYP2C19 2057 P-gp 1741	CYP1A1 389 CYP1A2 1197 CYP2A6 626 CYP2B6 776 CYP2C8 810 CYP2C19 1119 CYP2E1 729	CYP1A2 832 CYP2B6 669

Drug as perpetrator

Target	Modality	Activity
CYP2C19 2141	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000ClinPharmR.pdf#page=35 Page: 35.0	no [IC50 25 uM]
CYP1A2 2333	inducer 1966 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000ClinPharmR.pdf#page=40 Page: 40.0	no
CYP1A2 2333	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000ClinPharmR.pdf#page=35 Page: 35.0	no
CYP2A6 911	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000ClinPharmR.pdf#page=35 Page: 35.0	no
CYP2C8 1117	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000ClinPharmR.pdf#page=35 Page: 35.0	no
CYP2C9 2497	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000ClinPharmR.pdf#page=35 Page: 35.0	no
CYP2D6 2546	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000ClinPharmR.pdf#page=35 Page: 35.0	no
P-gp 1932	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000ClinPharmR.pdf#page=39 Page: 39.0	no
CYP2B6 1160	inducer 1966 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000ClinPharmR.pdf#page=40 Page: 40.0	yes
CYP3A4 2633	inducer 1966 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000ClinPharmR.pdf#page=40 Page: 40.0	yes
CYP3A4 2633	inhibitor 4027 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000ClinPharmR.pdf#page=35 Page: 35.0	yes

Drug as victim

Target	Modality	Activity
CYP3A4 1946	substrate 4014 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000ClinPharmR.pdf#page=35 Page: 35.0	major
CYP1A1 389	substrate 4014 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000ClinPharmR.pdf#page=39 Page: 39.0	no
CYP1A2 1197	substrate 4014 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000ClinPharmR.pdf#page=39 Page: 39.0	no
CYP2A6 626	substrate 4014 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000ClinPharmR.pdf#page=39 Page: 39.0	no
CYP2B6 776	substrate 4014 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000ClinPharmR.pdf#page=39 Page: 39.0	no
CYP2C19 1119	substrate 4014 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000ClinPharmR.pdf#page=39 Page: 39.0	no
CYP2C8 810	substrate 4014 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000ClinPharmR.pdf#page=39 Page: 39.0	no
CYP2C9 1193	substrate 4014 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000ClinPharmR.pdf#page=39 Page: 39.0	no
CYP2D6 1388	substrate 4014 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000ClinPharmR.pdf#page=39 Page: 39.0	no
CYP2E1 729	substrate 4014 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000ClinPharmR.pdf#page=39 Page: 39.0	no

Tox targets

Target	Modality	Activity	Metabolite	Clinical evidence
hERG 2263	inhibitor 2237 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/205598Orig1s000PharmR.pdf#page=28 Page: 28.0

Sourcing

Vendor/Aggregator	ID	URL
Chemieliva Pharmaceutical Co., Ltd	PBCM1293258
Compound Cloud	71526737
Compound Cloud	9804938
Hairui	HR139802	http://www.hairuichem.com/en/product/381231-18-1.html
iChemical	EBD3322128	http://www.ichemical.com/products/381231-18-1.html?utm_source=PubChem&utm_medium=website&utm_campaign=product%20catalogs
Lan Pharmatech	LAN-B38700
Smolecule	S534360	http://www.smolecule.com/products/s534360
Stork BioChemicals	4LSC2025
Synblock Inc	SB16909	http://www.synblock.com/product/381231-18-1.html
THE BioTek	bt-274659	https://www.thebiotek.com/product/inhibitors/bt-274659
THE BioTek	bt-339764	https://www.thebiotek.com/product/others/bt-339764
ZINC	ZINC000001554197	http://zinc15.docking.org/substances/ZINC000001554197

PubMed

Title	Date	PubMed
The macimorelin-stimulated growth hormone test for adult growth hormone deficiency diagnosis.	2014-07	24834478
Macimorelin (AEZS-130)-stimulated growth hormone (GH) test: validation of a novel oral stimulation test for the diagnosis of adult GH deficiency.	2013-06	23559086
Effect of food on the pharmacokinetics and pharmacodynamics of an oral ghrelin agonist (ARD-07) in healthy subjects.	2009-05	19293342

Patents

Sample Use Guides

In Vivo Use Guide

Recommended dose is 0.5 mg/kg as a single oral dose, after fasting for at least 8 hours

Route of Administration: Oral

In Vitro Use Guide

Macimorelin exhibited a high binding affinity to the cloned hGHS-R 1a receptor (IC50 value of 123 nM).

Substance Class	Chemical Created by `admin` on `Mon Mar 31 22:56:03 GMT 2025` Edited by `admin` on `Mon Mar 31 22:56:03 GMT 2025`
Record UNII	AQZ1003RMG
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

MACRILEN

Preferred Name

English

MACIMORELIN ACETATE

Official Name

English

EP-1572 ACETATE

Code

English

MACIMORELIN ACETATE [ORANGE BOOK]

Common Name

English

MACIMORELIN ACETATE [USAN]

Common Name

English

MACIMORELIN ACETATE [MI]

Common Name

English

Macimorelin acetate [WHO-DD]

Common Name

English

ARD-07 ACETATE

Code

English

N2-(2-AMINO-2-METHYLPROPANOYL-N1-((1R)-1-FORMAMIDO-2-(1H-INDOL-3-YL)ETHYL)- D-TRYPTOPHANAMIDE ACETATE

Common Name

English

D-TRYPTOPHANAMIDE, 2-METHYLALANYL-N-((1R)-1-(FORMYLAMINO)-2-(1H-INDOL-3-YL)ETHYL)-, ACETATE (1:1)

Systematic Name

English

D-87575 ACETATE

Code

English

Classification Tree Code System Code

FDA ORPHAN DRUG

225506