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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

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
Nonsense mutations: running the red light.
2005 Dec 8
Drug evaluation: PTC-124--a potential treatment of cystic fibrosis and Duchenne muscular dystrophy.
2006 Nov
Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers.
2007 Apr
Emerging drug treatments for cystic fibrosis.
2007 May
Chemical biology: ignore the nonsense.
2007 May 3
PTC124 targets genetic disorders caused by nonsense mutations.
2007 May 3
PTC124, nonsense mutations and Duchenne muscular dystrophy.
2007 Oct
Studies point way to new therapeutic prospects for muscular dystrophy.
2007 Sep 26
Pharmacological probing of type 1 autism.
2008 Aug
Ignoring the nonsense: a phase II trial in cystic fibrosis.
2008 Aug 30
Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial.
2008 Aug 30
New approaches to treatment of primary immunodeficiencies: fixing mutations with chemicals.
2008 Dec
Drug developers aim to treat cystic fibrosis through disease modification.
2008 Dec 15
Gunvalson decision sends shockwaves through industry.
2008 Nov
Introducing sense into nonsense in treatments of human genetic diseases.
2008 Nov
Reading through premature stop codons with PTC1 24. Project Catalyst to find more Duchenne drugs. Interview by Guenter Scheuerbrandt.
2008 Oct
Pharmaceuticals targeting nonsense mutations in genetic diseases: progress in development.
2009
PTC124 for cystic fibrosis.
2009 Apr 25
[Mutation-specific treatments for Duchenne muscular dystrophy].
2009 Aug
Update in cystic fibrosis 2008.
2009 Mar 15
Emerging genetic therapies to treat Duchenne muscular dystrophy.
2009 Oct
Emerging treatments in cystic fibrosis.
2009 Oct 1
UGA hopping: a sport for nephrologists too?
2010 Aug
Protein biomarkers in cystic fibrosis research: where next?
2010 Dec 16
Molecule of the month. Ataluren.
2010 Mar
Cystic fibrosis transmembrane conductance regulator protein repair as a therapeutic strategy in cystic fibrosis.
2010 Nov
Ataluren (PTC124) induces cystic fibrosis transmembrane conductance regulator protein expression and activity in children with nonsense mutation cystic fibrosis.
2010 Nov 15
[Research on mRNA degradation and drug discovery].
2010 Sep
Membrane blebbing as an assessment of functional rescue of dysferlin-deficient human myotubes via nonsense suppression.
2010 Sep
Read-through strategies for suppression of nonsense mutations in Duchenne/ Becker muscular dystrophy: aminoglycosides and ataluren (PTC124).
2010 Sep
A lack of premature termination codon read-through efficacy of PTC124 (Ataluren) in a diverse array of reporter assays.
2013
Orphan drug development in muscular dystrophy: update on two large clinical trials of dystrophin rescue therapies.
2013 Nov
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.
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
ACTIVE MOIETY
Structure of ATALUREN
METABOLITE (PARENT)
Structure of ATALUREN GLUCORONIDE
NIH
NCATS
PRIVACY ACT
ACCESSIBILITY
DISCLAIMER
HHS VULNERABILITY DISCLOSURE
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