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

Structure Search

SMILES

OC(=O)C1=CC(=CC=C1)C2=NOC(=N2)C3=C(F)C=CC=C3

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
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 18 Sources: http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Summary_for_the_public/human/002720/WC500171815.pdf	Primary		Approved 8583	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
Cystic fibrosis 120 Sources: http://www.ptcbio.com/en/pipeline/ataluren-translarna/	Primary		Phase III 665	Unknown Approved Use Unknown

C_max

Value	Dose	Analyte	Population
15.9 μg/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30629861/	5 mg/kg bw single, oral dose: 5 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: FASTED
13.5 μg/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30629861/	5 mg/kg bw single, oral dose: 5 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: FASTED
25.2 μg/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30629861/	10 mg/kg bw single, oral dose: 10 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: FASTED
29.9 μg/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30629861/	10 mg/kg bw single, oral dose: 10 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: FASTED
47.1 μg/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30629861/	20 mg/kg bw single, oral dose: 20 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: FASTED
48.8 μg/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30629861/	20 mg/kg bw single, oral dose: 20 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: FASTED
6 μg/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	3 mg/kg bw single, oral dose: 3 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FASTED
27 μg/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	10 mg/kg bw single, oral dose: 10 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FASTED
90 μg/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	30 mg/kg bw single, oral dose: 30 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FASTED
210 μg/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	100 mg/kg bw single, oral dose: 100 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FASTED
260 μg/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	150 mg/kg bw single, oral dose: 150 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FASTED
314 μg/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	200 mg/kg bw single, oral dose: 200 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FASTED
109 μg/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	50 mg/kg bw single, oral dose: 50 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FASTED
99 μg/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	50 mg/kg bw single, oral dose: 50 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: HIGH-FAT
16 μg/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	10 mg/kg bw 2 times / day multiple, oral dose: 10 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
13 μg/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	10 mg/kg bw 2 times / day multiple, oral dose: 10 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
26 μg/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	20 mg/kg bw 2 times / day multiple, oral dose: 20 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
32 μg/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	20 mg/kg bw 2 times / day multiple, oral dose: 20 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
41 μg/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	30 mg/kg bw 2 times / day multiple, oral dose: 30 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
50 μg/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	30 mg/kg bw 2 times / day multiple, oral dose: 30 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
59 μg/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	50 mg/kg bw 2 times / day multiple, oral dose: 50 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
80 μg/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	50 mg/kg bw 2 times / day multiple, oral dose: 50 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
81 μg/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	50 mg/kg bw 2 times / day multiple, oral dose: 50 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
65 μg/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	50 mg/kg bw 2 times / day multiple, oral dose: 50 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
53 μg/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	50 mg/kg bw 2 times / day multiple, oral dose: 50 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
85 μg/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	50 mg/kg bw 2 times / day multiple, oral dose: 50 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
76 μg/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	50 mg/kg bw 2 times / day multiple, oral dose: 50 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
76 μg/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	50 mg/kg bw 2 times / day multiple, oral dose: 50 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED

AUC

Value	Dose	Analyte	Population
49.3 μg × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30629861/	5 mg/kg bw single, oral dose: 5 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: FASTED
57.2 μg × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30629861/	5 mg/kg bw single, oral dose: 5 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: FASTED
104.5 μg × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30629861/	10 mg/kg bw single, oral dose: 10 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: FASTED
131.1 μg × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30629861/	10 mg/kg bw single, oral dose: 10 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: FASTED
228.5 μg × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30629861/	20 mg/kg bw single, oral dose: 20 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: FASTED
345.2 μg × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30629861/	20 mg/kg bw single, oral dose: 20 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: FASTED
27 μg × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	3 mg/kg bw single, oral dose: 3 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FASTED
110 μg × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	10 mg/kg bw single, oral dose: 10 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FASTED
482 μg × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	30 mg/kg bw single, oral dose: 30 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FASTED
2200 μg × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	100 mg/kg bw single, oral dose: 100 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FASTED
3001 μg × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	150 mg/kg bw single, oral dose: 150 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FASTED
2937 μg × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	200 mg/kg bw single, oral dose: 200 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FASTED
684 μg × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	50 mg/kg bw single, oral dose: 50 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FASTED
900 μg × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	50 mg/kg bw single, oral dose: 50 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: HIGH-FAT
91 μg × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	10 mg/kg bw 2 times / day multiple, oral dose: 10 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
81 μg × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	10 mg/kg bw 2 times / day multiple, oral dose: 10 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
164 μg × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	20 mg/kg bw 2 times / day multiple, oral dose: 20 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
179 μg × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	20 mg/kg bw 2 times / day multiple, oral dose: 20 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
314 μg × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	30 mg/kg bw 2 times / day multiple, oral dose: 30 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
330 μg × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	30 mg/kg bw 2 times / day multiple, oral dose: 30 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
415 μg × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	50 mg/kg bw 2 times / day multiple, oral dose: 50 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
574 μg × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	50 mg/kg bw 2 times / day multiple, oral dose: 50 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
551 μg × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	50 mg/kg bw 2 times / day multiple, oral dose: 50 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
463 μg × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	50 mg/kg bw 2 times / day multiple, oral dose: 50 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
359 μg × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	50 mg/kg bw 2 times / day multiple, oral dose: 50 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
688 μg × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	50 mg/kg bw 2 times / day multiple, oral dose: 50 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
644 μg × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	50 mg/kg bw 2 times / day multiple, oral dose: 50 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
540 μg × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	50 mg/kg bw 2 times / day multiple, oral dose: 50 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED

T_1/2

Value	Dose	Analyte	Population
3.2 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30629861/	5 mg/kg bw single, oral dose: 5 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: FASTED
3.2 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30629861/	5 mg/kg bw single, oral dose: 5 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: FASTED
3.3 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30629861/	10 mg/kg bw single, oral dose: 10 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: FASTED
3.4 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30629861/	10 mg/kg bw single, oral dose: 10 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: FASTED
3.4 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30629861/	20 mg/kg bw single, oral dose: 20 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: FASTED
3.4 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30629861/	20 mg/kg bw single, oral dose: 20 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: MALE food status: FASTED
3.1 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	3 mg/kg bw single, oral dose: 3 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FASTED
3.6 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	10 mg/kg bw single, oral dose: 10 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FASTED
4.7 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	30 mg/kg bw single, oral dose: 30 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FASTED
5.4 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	100 mg/kg bw single, oral dose: 100 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FASTED
5.1 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	150 mg/kg bw single, oral dose: 150 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FASTED
6.3 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	200 mg/kg bw single, oral dose: 200 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FASTED
5.3 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	50 mg/kg bw single, oral dose: 50 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FASTED
4.8 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	50 mg/kg bw single, oral dose: 50 mg/kg bw route of administration: Oral experiment type: SINGLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: HIGH-FAT
2.4 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	10 mg/kg bw 2 times / day multiple, oral dose: 10 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
2.5 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	10 mg/kg bw 2 times / day multiple, oral dose: 10 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
3.3 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	20 mg/kg bw 2 times / day multiple, oral dose: 20 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
3.5 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	20 mg/kg bw 2 times / day multiple, oral dose: 20 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
8.6 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	30 mg/kg bw 2 times / day multiple, oral dose: 30 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
4.7 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	30 mg/kg bw 2 times / day multiple, oral dose: 30 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
3.4 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	50 mg/kg bw 2 times / day multiple, oral dose: 50 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
5.3 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	50 mg/kg bw 2 times / day multiple, oral dose: 50 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
3.3 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	50 mg/kg bw 2 times / day multiple, oral dose: 50 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
6.3 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	50 mg/kg bw 2 times / day multiple, oral dose: 50 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
3.9 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	50 mg/kg bw 2 times / day multiple, oral dose: 50 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
6.8 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	50 mg/kg bw 2 times / day multiple, oral dose: 50 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
7.9 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	50 mg/kg bw 2 times / day multiple, oral dose: 50 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED
9 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/17389552/	50 mg/kg bw 2 times / day multiple, oral dose: 50 mg/kg bw route of administration: Oral experiment type: MULTIPLE co-administered:	ATALUREN plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE / MALE food status: FED

F_unbound

Value	Dose	Co-administered	Analyte	Population
0.4% EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/32196986/			ATALUREN plasma	Homo sapiens

Doses

Dose	Population	Adverse events
200 mg/kg single, oral Highest studied dose Dose: 200 mg/kg Route: oral Route: single Dose: 200 mg/kg Sources: https://pubmed.ncbi.nlm.nih.gov/17389552/	healthy, ADULT Health Status: healthy Age Group: ADULT Sex: M+F Food Status: FASTED Sources: https://pubmed.ncbi.nlm.nih.gov/17389552/	Other AEs: Headache, Dizziness... Other AEs: Headache Dizziness Nausea Vomiting Diarrhea Abdominal pain ALT increased (16.7%) AST increased (16.7%) Sources: https://pubmed.ncbi.nlm.nih.gov/17389552/
50 mg/kg 2 times / day multiple, oral Highest studied dose Dose: 50 mg/kg, 2 times / day Route: oral Route: multiple Dose: 50 mg/kg, 2 times / day Sources: https://pubmed.ncbi.nlm.nih.gov/17389552/	healthy, ADULT Health Status: healthy Age Group: ADULT Sex: M+F Food Status: FASTED Sources: https://pubmed.ncbi.nlm.nih.gov/17389552/	Other AEs: Vomiting, Headache... Other AEs: Vomiting Headache ALT increased (41.7%) AST increased (41.7%) Sources: https://pubmed.ncbi.nlm.nih.gov/17389552/

AEs

AE	Significance	Dose	Population
Abdominal pain		200 mg/kg single, oral Highest studied dose Dose: 200 mg/kg Route: oral Route: single Dose: 200 mg/kg Sources: https://pubmed.ncbi.nlm.nih.gov/17389552/	healthy, ADULT Health Status: healthy Age Group: ADULT Sex: M+F Food Status: FASTED Sources: https://pubmed.ncbi.nlm.nih.gov/17389552/
Diarrhea		200 mg/kg single, oral Highest studied dose Dose: 200 mg/kg Route: oral Route: single Dose: 200 mg/kg Sources: https://pubmed.ncbi.nlm.nih.gov/17389552/	healthy, ADULT Health Status: healthy Age Group: ADULT Sex: M+F Food Status: FASTED Sources: https://pubmed.ncbi.nlm.nih.gov/17389552/
Dizziness		200 mg/kg single, oral Highest studied dose Dose: 200 mg/kg Route: oral Route: single Dose: 200 mg/kg Sources: https://pubmed.ncbi.nlm.nih.gov/17389552/	healthy, ADULT Health Status: healthy Age Group: ADULT Sex: M+F Food Status: FASTED Sources: https://pubmed.ncbi.nlm.nih.gov/17389552/
Headache		200 mg/kg single, oral Highest studied dose Dose: 200 mg/kg Route: oral Route: single Dose: 200 mg/kg Sources: https://pubmed.ncbi.nlm.nih.gov/17389552/	healthy, ADULT Health Status: healthy Age Group: ADULT Sex: M+F Food Status: FASTED Sources: https://pubmed.ncbi.nlm.nih.gov/17389552/
Nausea		200 mg/kg single, oral Highest studied dose Dose: 200 mg/kg Route: oral Route: single Dose: 200 mg/kg Sources: https://pubmed.ncbi.nlm.nih.gov/17389552/	healthy, ADULT Health Status: healthy Age Group: ADULT Sex: M+F Food Status: FASTED Sources: https://pubmed.ncbi.nlm.nih.gov/17389552/
Vomiting		200 mg/kg single, oral Highest studied dose Dose: 200 mg/kg Route: oral Route: single Dose: 200 mg/kg Sources: https://pubmed.ncbi.nlm.nih.gov/17389552/	healthy, ADULT Health Status: healthy Age Group: ADULT Sex: M+F Food Status: FASTED Sources: https://pubmed.ncbi.nlm.nih.gov/17389552/
ALT increased	16.7%	200 mg/kg single, oral Highest studied dose Dose: 200 mg/kg Route: oral Route: single Dose: 200 mg/kg Sources: https://pubmed.ncbi.nlm.nih.gov/17389552/	healthy, ADULT Health Status: healthy Age Group: ADULT Sex: M+F Food Status: FASTED Sources: https://pubmed.ncbi.nlm.nih.gov/17389552/
AST increased	16.7%	200 mg/kg single, oral Highest studied dose Dose: 200 mg/kg Route: oral Route: single Dose: 200 mg/kg Sources: https://pubmed.ncbi.nlm.nih.gov/17389552/	healthy, ADULT Health Status: healthy Age Group: ADULT Sex: M+F Food Status: FASTED Sources: https://pubmed.ncbi.nlm.nih.gov/17389552/
Headache		50 mg/kg 2 times / day multiple, oral Highest studied dose Dose: 50 mg/kg, 2 times / day Route: oral Route: multiple Dose: 50 mg/kg, 2 times / day Sources: https://pubmed.ncbi.nlm.nih.gov/17389552/	healthy, ADULT Health Status: healthy Age Group: ADULT Sex: M+F Food Status: FASTED Sources: https://pubmed.ncbi.nlm.nih.gov/17389552/
Vomiting		50 mg/kg 2 times / day multiple, oral Highest studied dose Dose: 50 mg/kg, 2 times / day Route: oral Route: multiple Dose: 50 mg/kg, 2 times / day Sources: https://pubmed.ncbi.nlm.nih.gov/17389552/	healthy, ADULT Health Status: healthy Age Group: ADULT Sex: M+F Food Status: FASTED Sources: https://pubmed.ncbi.nlm.nih.gov/17389552/
ALT increased	41.7%	50 mg/kg 2 times / day multiple, oral Highest studied dose Dose: 50 mg/kg, 2 times / day Route: oral Route: multiple Dose: 50 mg/kg, 2 times / day Sources: https://pubmed.ncbi.nlm.nih.gov/17389552/	healthy, ADULT Health Status: healthy Age Group: ADULT Sex: M+F Food Status: FASTED Sources: https://pubmed.ncbi.nlm.nih.gov/17389552/
AST increased	41.7%	50 mg/kg 2 times / day multiple, oral Highest studied dose Dose: 50 mg/kg, 2 times / day Route: oral Route: multiple Dose: 50 mg/kg, 2 times / day Sources: https://pubmed.ncbi.nlm.nih.gov/17389552/	healthy, ADULT Health Status: healthy Age Group: ADULT Sex: M+F Food Status: FASTED Sources: https://pubmed.ncbi.nlm.nih.gov/17389552/

PubMed

Title	Date	PubMed
Orphan drug development in muscular dystrophy: update on two large clinical trials of dystrophin rescue therapies.	2013-11	24229740
A lack of premature termination codon read-through efficacy of PTC124 (Ataluren) in a diverse array of reporter assays.	2013	23824517
Protein biomarkers in cystic fibrosis research: where next?	2010-12-16	21167082
Ataluren (PTC124) induces cystic fibrosis transmembrane conductance regulator protein expression and activity in children with nonsense mutation cystic fibrosis.	2010-11-15	20622033
Cystic fibrosis transmembrane conductance regulator protein repair as a therapeutic strategy in cystic fibrosis.	2010-11	20829696
[Research on mRNA degradation and drug discovery].	2010-09	20838017
Membrane blebbing as an assessment of functional rescue of dysferlin-deficient human myotubes via nonsense suppression.	2010-09	20558759
Read-through strategies for suppression of nonsense mutations in Duchenne/ Becker muscular dystrophy: aminoglycosides and ataluren (PTC124).	2010-09	20519671
UGA hopping: a sport for nephrologists too?	2010-08	20501459
Molecule of the month. Ataluren.	2010-03	20369078
Emerging treatments in cystic fibrosis.	2009-10-01	19747007
Emerging genetic therapies to treat Duchenne muscular dystrophy.	2009-10	19745732
[Mutation-specific treatments for Duchenne muscular dystrophy].	2009-08	19697880
PTC124 for cystic fibrosis.	2009-04-25	19394530
Update in cystic fibrosis 2008.	2009-03-15	19264980
Pharmaceuticals targeting nonsense mutations in genetic diseases: progress in development.	2009	19627168
Drug developers aim to treat cystic fibrosis through disease modification.	2008-12-15	19052266
New approaches to treatment of primary immunodeficiencies: fixing mutations with chemicals.	2008-12	18978469
Gunvalson decision sends shockwaves through industry.	2008-11	18997740
Introducing sense into nonsense in treatments of human genetic diseases.	2008-11	18937996
Reading through premature stop codons with PTC1 24. Project Catalyst to find more Duchenne drugs. Interview by Guenter Scheuerbrandt.	2008-10	19364064
Ignoring the nonsense: a phase II trial in cystic fibrosis.	2008-08-30	18722009
Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial.	2008-08-30	18722008
Pharmacological probing of type 1 autism.	2008-08	18512134
PTC124, nonsense mutations and Duchenne muscular dystrophy.	2007-10	17952895
Studies point way to new therapeutic prospects for muscular dystrophy.	2007-09-26	17895449
Chemical biology: ignore the nonsense.	2007-05-03	17450128
PTC124 targets genetic disorders caused by nonsense mutations.	2007-05-03	17450125
Emerging drug treatments for cystic fibrosis.	2007-05	17604505
Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers.	2007-04	17389552
Drug evaluation: PTC-124--a potential treatment of cystic fibrosis and Duchenne muscular dystrophy.	2006-11	17096300
Nonsense mutations: running the red light.	2005-12-08	16340982

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.

Substance Class	Chemical Created by `admin` on `Mon Mar 31 18:20:07 GMT 2025` Edited by `admin` on `Mon Mar 31 18:20:07 GMT 2025`
Record UNII	K16AME9I3V
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

ATALUREN

Official Name

English

TRANSLARNA

Preferred 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

Classification Tree Code System Code

EU-Orphan Drug

EU/3/15/1561