Metyrosine

Summary

Metyrosine (CHEMBL1200862) is an approved small-molecule antihypertensive agent (ATC C02KB01) targeting TH; indicated across 3 conditions including hypertensive disorder and adrenal gland pheochromocytoma.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: C02KB01
• Targets: 1 (TH)
• Indications: 3 conditions
• Clinical trials: 2
• Chemistry: 195.21 Da · C10H13NO3

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1200862
Name	Metyrosine
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	441350
ChEBI	CHEBI:6912
ATC	C02KB01
Molecular formula	C10H13NO3
Molecular weight	195.21
InChIKey	NHTGHBARYWONDQ-JTQLQIEISA-N

SMILES: C[C@](CC1=CC=C(C=C1)O)(C(=O)O)N

IUPAC name: (2S)-2-amino-3-(4-hydroxyphenyl)-2-methylpropanoic acid

ChEBI definition: An L-tyrosine derivative that consists of L-tyrosine bearing an additional methyl substituent at position 2. An inhibitor of the enzyme tyrosine 3-monooxygenase, and consequently of the synthesis of catecholamines. It is used to control the symptoms of excessive sympathetic stimulation in patients with pheochromocytoma.

Pharmacological roles (ChEBI): antihypertensive agent, EC 1.14.16.2 (tyrosine 3-monooxygenase) inhibitor.

Also known as: Demser, L-tyrosine, Methyltyrosine, Metirosina, Metirosine, Metyrosine, MK-781, Racemetirosine, (s)-, SID50106644, METYROSINE, SID90341393, SID29215162

Patent coverage: 2,311 distinct patent families (8,501 SureChEMBL compound mentions), from 1 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).

Gene	Target	Action	pAffinity	Cancer dependency	UniProt
TH	L-Tyrosine hydroxylase	Inhibition		0.2%	P07101

Broader ChEMBL bioactivity targets: 2 (assay-derived). Sample: RecQ-like DNA helicase BLM, Inositol monophosphatase 1.

Bioactivity

ChEMBL activities: 3 potent at pChembl ≥ 5 of 3 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
P97697	5.4	Potency	3981	nM	CHEMBL_ACT_4408662
BLM	5.25	Potency	5623	nM	CHEMBL_ACT_4753848
BLM	5.25	Potency	5623	nM	CHEMBL_ACT_4941900

Target pathways

Aggregated over 1 target gene(s): TH.

Top Reactome pathways

1 total, by targets touching each:

Pathway	Targets	Genes
Catecholamine biosynthesis	1	TH

Dominant GO biological processes

GO term	Targets
response to hypoxia	1
synaptic transmission, dopaminergic	1
heart morphogenesis	1
dopamine biosynthetic process from tyrosine	1
heart development	1
visual perception	1
learning	1
memory	1
mating behavior	1
locomotory behavior	1
regulation of heart contraction	1
anatomical structure morphogenesis	1
animal organ morphogenesis	1
dopamine biosynthetic process	1
epinephrine biosynthetic process	1

Indications & clinical

Indications

3 indications (2 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
hypertensive disorder	4	MONDO:0005044	EFO:0000537
adrenal gland pheochromocytoma	4	MONDO:0004974	EFO:0000239
psychotic disorder	2	MONDO:0005485	EFO:0005407

Clinical trials

Total trials: 2.

Phase distribution

Phase	Trials
PHASE2	1
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT01127503	PHASE2	TERMINATED	Metyrosine (Demser®) for the Treatment of Psychotic Disorders in Patients With Velocardiofacial Syndrome
NCT02179814	Not specified	SUSPENDED	Neural Response to Catecholamine Depletion in Subjects Suffering From Bulimia Nervosa in Their Past and Healthy Controls

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).