Ataluren

Suppresses premature stop mutation

Phase of research

EMA-approved for Other Purpose

How it helps

Restore CFTR Function

General information

Ataluren is a pharmaceutical drug. It was approved in the European Union in 2014 for the treatment of Duchenne muscular dystrophy and it is sold under the name Translarna™. It does not yet have approval from the FDA. Ataluren targets nonsense mutations, where it is able to skip and produce full-length protein. In relation to cystic fibrosis, our AIM tool found the data that ataluren can be beneficial for patients with nonsense mutations who are not treated with tobramycin.

Ataluren on PubChem
Ataluren on DrugBank
Ataluren on Wikipedia

Synonyms

PTC124

Marketed as

Dietary sources

N/A

Structure not available

C15H9FN2O3

Drug-Mutation Relation

Treats

Mutation Number of sources Average impact factor
G542X
1 3.48
Link Tested on Impact factor Notes
A randomized placebo-controlled trial of ataluren for the treatment of nonsense mutation cystic fibrosis humans 3.48 Effective only in patients not using tobramycin.
R1162X
1 3.48
Link Tested on Impact factor Notes
A randomized placebo-controlled trial of ataluren for the treatment of nonsense mutation cystic fibrosis humans 3.48 Effective only in patients not using tobramycin.
R553X
1 3.48
Link Tested on Impact factor Notes
A randomized placebo-controlled trial of ataluren for the treatment of nonsense mutation cystic fibrosis humans 3.48 Effective only in patients not using tobramycin.
W1282X
1 3.48
Link Tested on Impact factor Notes
A randomized placebo-controlled trial of ataluren for the treatment of nonsense mutation cystic fibrosis humans 3.48 Effective only in patients not using tobramycin.

Does not treat

Mutation Number of sources Average impact factor
nonsense mutations
2 3.48
Link Tested on Impact factor Notes
Phase 3 study of Ataluren in people with CF who have a nonsense mutation and are not taking aminoglycosides (PTC124-GD-021-CF) humans N/A
A randomized placebo-controlled trial of ataluren for the treatment of nonsense mutation cystic fibrosis humans 3.48 Might be beneficial for patients not using tobramycin.
Link Tested on Impact factor Notes
Phase 3 study of Ataluren in people with CF who have a nonsense mutation and are not taking aminoglycosides (PTC124-GD-021-CF) humans N/A
A randomized placebo-controlled trial of ataluren for the treatment of nonsense mutation cystic fibrosis humans 3.48 Might be beneficial for patients not using tobramycin.