ATALUREN

Possibly Marketed Outside US

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

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	MOL RATIO 1 MOL RATIO (average)

Stereochemistry	ACHIRAL
Additional Stereochemistry	No
Defined Stereocenters	0 / 0
E/Z Centers	0
Optical Activity	NONE

Description

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	Interacts 323

Conditions

Condition	Modality	Targets	Highest Phase	Product
Duchenne muscular dystrophy 19	Primary		Approved 8,429	TRANSLARNA
Cystic fibrosis 120	Primary		Phase III 656	Unknown

C_max

Value	Dose	Analyte	Population
15.9 μg/mL	5 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
13.5 μg/mL	5 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
25.2 μg/mL	10 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
29.9 μg/mL	10 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
47.1 μg/mL	20 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
48.8 μg/mL	20 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
6 μg/mL	3 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
27 μg/mL	10 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
90 μg/mL	30 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
210 μg/mL	100 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
260 μg/mL	150 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
314 μg/mL	200 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
109 μg/mL	50 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
99 μg/mL	50 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
16 μg/mL	10 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
13 μg/mL	10 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
26 μg/mL	20 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
32 μg/mL	20 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
41 μg/mL	30 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
50 μg/mL	30 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
59 μg/mL	50 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
80 μg/mL	50 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
81 μg/mL	50 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
65 μg/mL	50 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
53 μg/mL	50 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
85 μg/mL	50 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
76 μg/mL	50 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
76 μg/mL	50 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens

AUC

Value	Dose	Analyte	Population
49.3 μg × h/mL	5 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
57.2 μg × h/mL	5 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
104.5 μg × h/mL	10 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
131.1 μg × h/mL	10 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
228.5 μg × h/mL	20 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
345.2 μg × h/mL	20 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
27 μg × h/mL	3 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
110 μg × h/mL	10 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
482 μg × h/mL	30 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
2200 μg × h/mL	100 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
3001 μg × h/mL	150 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
2937 μg × h/mL	200 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
684 μg × h/mL	50 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
900 μg × h/mL	50 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
91 μg × h/mL	10 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
81 μg × h/mL	10 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
164 μg × h/mL	20 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
179 μg × h/mL	20 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
314 μg × h/mL	30 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
330 μg × h/mL	30 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
415 μg × h/mL	50 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
574 μg × h/mL	50 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
551 μg × h/mL	50 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
463 μg × h/mL	50 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
359 μg × h/mL	50 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
688 μg × h/mL	50 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
644 μg × h/mL	50 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
540 μg × h/mL	50 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens

T_1/2

Value	Dose	Analyte	Population
3.2 h	5 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
3.2 h	5 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
3.3 h	10 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
3.4 h	10 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
3.4 h	20 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
3.4 h	20 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
3.1 h	3 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
3.6 h	10 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
4.7 h	30 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
5.4 h	100 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
5.1 h	150 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
6.3 h	200 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
5.3 h	50 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
4.8 h	50 mg/kg bw single, oral	ATALUREN plasma	Homo sapiens
2.4 h	10 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
2.5 h	10 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
3.3 h	20 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
3.5 h	20 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
8.6 h	30 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
4.7 h	30 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
3.4 h	50 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
5.3 h	50 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
3.3 h	50 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
6.3 h	50 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
3.9 h	50 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
6.8 h	50 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
7.9 h	50 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens
9 h	50 mg/kg bw 2 times / day multiple, oral	ATALUREN plasma	Homo sapiens

F_unbound

Value	Dose	Co-administered	Analyte	Population
0.4%			ATALUREN plasma	Homo sapiens

Doses

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Dose	Population	Adverse events
200 mg/kg single, oral Highest studied dose	healthy, ADULT	Other AEs: Headache, Dizziness...
50 mg/kg 2 times / day multiple, oral Highest studied dose	healthy, ADULT	Other AEs: Vomiting, Headache...

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AEs

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AE	Dose	Population
Abdominal pain	200 mg/kg single, oral Highest studied dose	healthy, ADULT
Diarrhea	200 mg/kg single, oral Highest studied dose	healthy, ADULT
Dizziness	200 mg/kg single, oral Highest studied dose	healthy, ADULT
Headache	200 mg/kg single, oral Highest studied dose	healthy, ADULT
Nausea	200 mg/kg single, oral Highest studied dose	healthy, ADULT

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PubMed

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Title	Date	PubMed
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	17389552
Emerging drug treatments for cystic fibrosis.	2007 May	17604505
Ignoring the nonsense: a phase II trial in cystic fibrosis.	2008 Aug 30	18722009
Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial.	2008 Aug 30	18722008
New approaches to treatment of primary immunodeficiencies: fixing mutations with chemicals.	2008 Dec	18978469

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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
Record UNII	K16AME9I3V
Record Status	`Validated (UNII)`
Record Version

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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

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Classification Tree

Code System

Code

Positive

EU-Orphan Drug

EU/3/15/1561

Designated/Withdrawn

FDA ORPHAN DRUG

192004

Designated/Withdrawn

FDA ORPHAN DRUG

255907

Designated/Withdrawn

FDA ORPHAN DRUG

490215

Other drugs for disorders of the musculo-skeletal system

WHO-ATC

M09AX03

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Code System

Code

Type

Description

ChEMBL

CHEMBL256997

PRIMARY

EVMPD

SUB89249

PRIMARY

DRUG BANK

DB05016

PRIMARY

EPA CompTox

DTXSID5046776

PRIMARY

EU-Orphan Drug

EU/3/05/278(POSITIVE)

PRIMARY

treatment of Duchenne muscular dystrophy

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Related Record

Type

Details


					K16AME9I3V

ATALUREN

EXCRETED UNCHANGED

Qualification:

URINE

Amount:

0.83 % (average)
of dose excreted


					K16AME9I3V

ATALUREN

EXCRETED UNCHANGED

Qualification:

FECAL

Amount:

0.4 % (average)
of dose excreted

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Related Record

Type

Details


					4HDD4R2AEW

3-(AMINOIMINOMETHYL)BENZOIC ACID

METABOLITE -> PARENT

Qualification:

FECAL

Amount:

36.6 % (average)
of dose excreted


					8U8VEL1T75

OXADIAZOLE RING OPENED ATALUREN

METABOLITE -> PARENT

Qualification:

URINE

Amount:

0.16 % (average)
of dose excreted


					4GR4LS9SWP

ATALUREN GLUCORONIDE

METABOLITE -> PARENT

Interaction Type:

MAJOR

Qualification:

PLASMA

Amount:

7.6 % (average)
of plasma AUC of ataluren


					4GR4LS9SWP

ATALUREN GLUCORONIDE

METABOLITE -> PARENT

Interaction Type:

MAJOR

Qualification:

URINE

Amount:

48.6 % (average)
of dose excreted


					4HDD4R2AEW

3-(AMINOIMINOMETHYL)BENZOIC ACID

METABOLITE -> PARENT

Qualification:

URINE

Amount:

1.98 % (average)
of dose excreted

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Related Record

Type

Details


					K16AME9I3V

ATALUREN

ACTIVE MOIETY

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Name	Property Type	Amount	Parameters
Biological Half-life	PHARMACOKINETIC	6.4 hours (average)	ORAL ADMINISTRATION

Tmax	PHARMACOKINETIC	1 hours (average)	ORAL ADMINISTRATION

SMILES

InChI

CNS Activity

Originator

Approval Year

Targets

Conditions

Cmax

AUC

T1/2

Funbound

Doses

AEs

PubMed

Patents

Sample Use Guides

C_max

T_1/2

F_unbound