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Details

Stereochemistry ACHIRAL
Molecular Formula C15H9FN2O3
Molecular Weight 284.242
Optical Activity NONE
Defined Stereocenters 0 / 0
E/Z Centers 0
Charge 0

SHOW SMILES / InChI
Structure of ATALUREN

SMILES

OC(=O)C1=CC(=CC=C1)C2=NOC(=N2)C3=CC=CC=C3F

InChI

InChIKey=OOUGLTULBSNHNF-UHFFFAOYSA-N
InChI=1S/C15H9FN2O3/c16-12-7-2-1-6-11(12)14-17-13(18-21-14)9-4-3-5-10(8-9)15(19)20/h1-8H,(H,19,20)

HIDE SMILES / InChI
Molecular Formula C15H9FN2O3
Molecular Weight 284.242
Charge 0
Count
Stereochemistry ACHIRAL
Additional Stereochemistry No
Defined Stereocenters 0 / 0
E/Z Centers 0
Optical Activity NONE

Description
Curator's Comment: description was created based on several sources, including http://www.ptcbio.com/en/pipeline/ataluren-translarna/

Ataluren (Translarna) is a small-molecule drug approved in Europe for the treatment of Duchenne muscular dystrophy caused by a nonsense mutation. Ataluren interacts with the ribosome enabling it to read through premature nonsense stop signals on mRNA and allowing the cell to produce a full-length, functional protein. Ataluren is also being tested in phase III for cystic fibrosis caused by a nonsense mutation.

CNS Activity

Originator

Approval Year

Unknown
139594
Targets

Targets

Primary TargetPharmacologyConditionPotency
Interacts
323
Conditions

Conditions

ConditionModalityTargetsHighest PhaseProduct
Primary
Approved
8429
TRANSLARNA

Approved Use

Translarna is a medicine that contains the active substance ataluren. It is used to treat patients aged 5 years and older with Duchenne muscular dystrophy who are able to walk.

Launch Date

2014
Primary
Phase III
656
Unknown

Approved Use

Unknown
PubMed

PubMed

TitleDatePubMed
PTC124 targets genetic disorders caused by nonsense mutations.
2007 May 3
PTC124, nonsense mutations and Duchenne muscular dystrophy.
2007 Oct
New approaches to treatment of primary immunodeficiencies: fixing mutations with chemicals.
2008 Dec
Gunvalson decision sends shockwaves through industry.
2008 Nov
Reading through premature stop codons with PTC1 24. Project Catalyst to find more Duchenne drugs. Interview by Guenter Scheuerbrandt.
2008 Oct
[Mutation-specific treatments for Duchenne muscular dystrophy].
2009 Aug
Update in cystic fibrosis 2008.
2009 Mar 15
Protein biomarkers in cystic fibrosis research: where next?
2010 Dec 16
Patents

Patents

06992096
2
07202262
1
07304080
1
07419991
1
07683082
1
07772259
2
07902235
1
08017636
1
08129540
2
08163782
2
08227494
1
08299105
1
08486982
1
08710031
1
08716321
1
08796322
1
20040204461
1
20050164973
1
20060035943
1
20060148863
1
20060148864
1
20070161687
1
20080114039
1
20080139818
1
20080171377
1
20090093418
1
20090149513
1
20090203752
1
20100063297
1
JP2013189465A
1

Sample Use Guides

In Vivo Use Guide
Ataluren (Translarna) is taken three times a day, and the recommended dose is 10 mg/kg (10 mg per kilogram body weight) in the morning, 10 mg/kg at midday and 20 mg/kg in the evening (making a total daily dose of 40 mg/kg). Granules should be mixed with liquid or semi-solid food (such as yogurt).
Route of Administration: Oral
In Vitro Use Guide
Patient-dervied myotubes cultured with ataluren (10 ug/mL) resulted in the expression of functional dysferlin protein in myotubes derived from a nonsense mutation Miyoshi myopathy patient.
Substance Class Chemical
Created
by admin
on Fri Dec 15 16:08:43 GMT 2023
Edited
by admin
on Fri Dec 15 16:08:43 GMT 2023
Record UNII
K16AME9I3V
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
ATALUREN
DASH   INN   USAN   WHO-DD  
USAN   INN  
Official Name English
ATALUREN [MI]
Common Name English
3-[5-(2-Fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid
Systematic Name English
Ataluren [WHO-DD]
Common Name English
BENZOIC ACID, 3-(5-(2-FLUOROPHENYL)-1,2,4-OXADIAZOL-3-YL)-
Common Name English
ataluren [INN]
Common Name English
PTC-124
Code English
PTC124
Code English
ATALUREN [USAN]
Common Name English
TRANSLARNA
Brand Name English
Classification Tree Code System Code
EU-Orphan Drug EU/3/15/1561
Created by admin on Fri Dec 15 16:08:43 GMT 2023 , Edited by admin on Fri Dec 15 16:08:43 GMT 2023
FDA ORPHAN DRUG 192004
Created by admin on Fri Dec 15 16:08:43 GMT 2023 , Edited by admin on Fri Dec 15 16:08:43 GMT 2023
FDA ORPHAN DRUG 255907
Created by admin on Fri Dec 15 16:08:43 GMT 2023 , Edited by admin on Fri Dec 15 16:08:43 GMT 2023
FDA ORPHAN DRUG 490215
Created by admin on Fri Dec 15 16:08:43 GMT 2023 , Edited by admin on Fri Dec 15 16:08:43 GMT 2023
WHO-ATC M09AX03
Created by admin on Fri Dec 15 16:08:43 GMT 2023 , Edited by admin on Fri Dec 15 16:08:43 GMT 2023
FDA ORPHAN DRUG 197204
Created by admin on Fri Dec 15 16:08:43 GMT 2023 , Edited by admin on Fri Dec 15 16:08:43 GMT 2023
FDA ORPHAN DRUG 455814
Created by admin on Fri Dec 15 16:08:43 GMT 2023 , Edited by admin on Fri Dec 15 16:08:43 GMT 2023
EMA ASSESSMENT REPORTS TRANSLARNA (AUTHORIZED: MUSCULAR DYSTROPHY, DUCHENNE)
Created by admin on Fri Dec 15 16:08:43 GMT 2023 , Edited by admin on Fri Dec 15 16:08:43 GMT 2023
EU-Orphan Drug EU/3/14/1380
Created by admin on Fri Dec 15 16:08:43 GMT 2023 , Edited by admin on Fri Dec 15 16:08:43 GMT 2023
Code System Code Type Description
ChEMBL
CHEMBL256997
Created by admin on Fri Dec 15 16:08:43 GMT 2023 , Edited by admin on Fri Dec 15 16:08:43 GMT 2023
PRIMARY
EVMPD
SUB89249
Created by admin on Fri Dec 15 16:08:43 GMT 2023 , Edited by admin on Fri Dec 15 16:08:43 GMT 2023
PRIMARY
DRUG BANK
DB05016
Created by admin on Fri Dec 15 16:08:43 GMT 2023 , Edited by admin on Fri Dec 15 16:08:43 GMT 2023
PRIMARY
EPA CompTox
DTXSID5046776
Created by admin on Fri Dec 15 16:08:43 GMT 2023 , Edited by admin on Fri Dec 15 16:08:43 GMT 2023
PRIMARY
EU-Orphan Drug
EU/3/05/278(POSITIVE)
Created by admin on Fri Dec 15 16:08:43 GMT 2023 , Edited by admin on Fri Dec 15 16:08:43 GMT 2023
PRIMARY treatment of Duchenne muscular dystrophy
MERCK INDEX
m11736
Created by admin on Fri Dec 15 16:08:43 GMT 2023 , Edited by admin on Fri Dec 15 16:08:43 GMT 2023
PRIMARY
FDA UNII
K16AME9I3V
Created by admin on Fri Dec 15 16:08:43 GMT 2023 , Edited by admin on Fri Dec 15 16:08:43 GMT 2023
PRIMARY
PUBCHEM
11219835
Created by admin on Fri Dec 15 16:08:43 GMT 2023 , Edited by admin on Fri Dec 15 16:08:43 GMT 2023
PRIMARY
IUPHAR
7341
Created by admin on Fri Dec 15 16:08:43 GMT 2023 , Edited by admin on Fri Dec 15 16:08:43 GMT 2023
PRIMARY
CAS
775304-57-9
Created by admin on Fri Dec 15 16:08:43 GMT 2023 , Edited by admin on Fri Dec 15 16:08:43 GMT 2023
PRIMARY
SMS_ID
100000140052
Created by admin on Fri Dec 15 16:08:43 GMT 2023 , Edited by admin on Fri Dec 15 16:08:43 GMT 2023
PRIMARY
WIKIPEDIA
ATALUREN
Created by admin on Fri Dec 15 16:08:43 GMT 2023 , Edited by admin on Fri Dec 15 16:08:43 GMT 2023
PRIMARY
USAN
TT-04
Created by admin on Fri Dec 15 16:08:43 GMT 2023 , Edited by admin on Fri Dec 15 16:08:43 GMT 2023
PRIMARY
DRUG CENTRAL
4872
Created by admin on Fri Dec 15 16:08:43 GMT 2023 , Edited by admin on Fri Dec 15 16:08:43 GMT 2023
PRIMARY
NCI_THESAURUS
C169791
Created by admin on Fri Dec 15 16:08:43 GMT 2023 , Edited by admin on Fri Dec 15 16:08:43 GMT 2023
PRIMARY
INN
8956
Created by admin on Fri Dec 15 16:08:43 GMT 2023 , Edited by admin on Fri Dec 15 16:08:43 GMT 2023
PRIMARY
Related Record Type Details
Structure of ATALUREN
EXCRETED UNCHANGED
URINE
Structure of ATALUREN
EXCRETED UNCHANGED
FECAL
Related Record Type Details
Structure of 3-(AMINOIMINOMETHYL)BENZOIC ACID
METABOLITE -> PARENT
FECAL
Structure of OXADIAZOLE RING OPENED ATALUREN
METABOLITE -> PARENT
URINE
Structure of ATALUREN GLUCORONIDE
METABOLITE -> PARENT
MAJOR
PLASMA
Structure of ATALUREN GLUCORONIDE
METABOLITE -> PARENT
MAJOR
URINE
Structure of 3-(AMINOIMINOMETHYL)BENZOIC ACID
METABOLITE -> PARENT
URINE
Structure of OXADIAZOLE RING OPENED ATALUREN
METABOLITE -> PARENT
FECAL
Related Record Type Details
Structure of ATALUREN
ACTIVE MOIETY
Name Property Type Amount Referenced Substance Defining Parameters References
Biological Half-life PHARMACOKINETIC ORAL ADMINISTRATION

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