ATALUREN

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 Search

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
Sources: http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Summary_for_the_public/human/002720/WC500171815.pdf
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

Targets

Primary Target	Pharmacology	Condition	Potency
Ribosome 11 Target ID: CHEMBL613299 Sources: http://www.ptcbio.com/en/pipeline/ataluren-translarna/	Interacts 323

Conditions

Condition	Modality	Targets	Highest Phase	Product
Duchenne muscular dystrophy 19 Sources: http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Summary_for_the_public/human/002720/WC500171815.pdf	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 1.40676484E12
Cystic fibrosis 120 Sources: http://www.ptcbio.com/en/pipeline/ataluren-translarna/	Primary		Phase III 656	Unknown Approved Use Unknown

PubMed

Title	Date	PubMed
Drug evaluation: PTC-124--a potential treatment of cystic fibrosis and Duchenne muscular dystrophy.	2006 Nov	17096300
Emerging drug treatments for cystic fibrosis.	2007 May	17604505
Membrane blebbing as an assessment of functional rescue of dysferlin-deficient human myotubes via nonsense suppression.	2010 Sep	20558759

Patents

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

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