Compound Detail
CHEMBL256997
ATALUREN 💊 Approved Drug
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💊 Approved Drug
Basic Information
| ChEMBL ID | CHEMBL256997 |
| Name | ATALUREN |
| Phase | Approved |
| Structure Type | MOL |
| InChI Key | OOUGLTULBSNHNF-UHFFFAOYSA-N |
| Therapeutic | ✓ Yes |
3
Targets
5
Activities
3
Assay Types
2
PK Parameters
20
Publications
5
Known Names
7
Indications
1
Mechanisms
Molecular Properties
284.2
MW (Da)
3.24
ALogP
4
HBA
1
HBD
76.2
PSA (Ų)
0.80
QED
0
Ro5 Violations
3
Rot. Bonds
Drug Information
| Molecule Type | Small molecule |
| First Approval | 2014 |
| Availability | Unknown |
| Chirality | Achiral |
Mechanism of Action
| Mechanism | Action Type | Target | Direct | Efficacy |
|---|---|---|---|---|
| 80S Ribosome modulator | MODULATOR | 80S Ribosome | ✓ | ✓ |
Indications
Muscular Dystrophy, Duchenne Muscular Dystrophy, Duchenne Cystic Fibrosis Aniridia Epilepsy Hemophilia A Hemophilia B
ATC Classification
M09AX03
ataluren
Level 1: MUSCULO-SKELETAL SYSTEM
Level 2: OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM
Activity Summary
EC50 2 meas. best 5.96
Kd 2 meas. best 6.7
IC50 1 meas. best 6.52
Target Activity Profile
| Target | Type | Best pChEMBL | Mean pChEMBL | Best (nM) | # Data |
|---|---|---|---|---|---|
| Flavin reductase (NADPH) CHEMBL5019 | Kd | 6.7 | 6.7 | 199.5 | 2 |
| Histone deacetylase 6 CHEMBL1865 | IC50 | 6.52 | 6.52 | 300.9 | 1 |
| Unchecked CHEMBL612545 | EC50 | 5.96 | 5.96 | 1100.0 | 1 |
Pharmacokinetic Data
| Parameter | Value | Units | Species |
|---|---|---|---|
| T1/2 | 5.0 | hr | Homo sapiens |
| T1/2 | 4.22 | hr | Mus musculus |
Activity Data Samples
Top measurements by pChEMBL value
| Target | Type | Rel. | Value | Units | pChEMBL | Assay | PubMed |
|---|---|---|---|---|---|---|---|
| Flavin reductase (NADPH) | Kd | = | 200.0 | nM | 6.7 | Binding affinity to human N-terminal His6-tagged and thrombin cleavage fused BLV... | 34957824 |
| Flavin reductase (NADPH) | Kd | = | 199.53 | nM | 6.7 | Binding affinity to human N-terminal His6-tagged and thrombin cleavage fused BLV... | 34957824 |
| Histone deacetylase 6 | IC50 | = | 300.9 | nM | 6.52 | Determination of IC50 values for inhibition of enzymatic assay of human HDAC6 wi... | - |
| Unchecked | EC50 | = | 1100.0 | nM | 5.96 | Activation of NF-KappaB (unknown origin) expressed in SH-SY5Y cells co-transfect... | 33139114 |
Literature References
Data from ChEMBL 36 via Core Engine