Ataluren
Translarna (ataluren) is a small molecule pharmaceutical. Ataluren was first approved as Translarna on 2014-07-31. It has been approved in Europe to treat duchenne muscular dystrophy.
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Commercial
Therapeutic Areas
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Indications
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Indication | Ontology | MeSH | ICD-10 |
|---|---|---|---|
| duchenne muscular dystrophy | EFO_0000429 | D020388 | — |
Agency Specific
FDA
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Patent Expiration
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Clinical
Clinical Trials
34 clinical trials
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Indications Phases 4
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Fibrosis | D005355 | — | — | — | 4 | 5 | 2 | — | 11 |
| Cystic fibrosis | D003550 | EFO_0000390 | E84 | — | 4 | 5 | 2 | — | 11 |
Indications Phases 3
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Duchenne muscular dystrophy | D020388 | EFO_0000429 | — | — | 9 | 5 | — | 2 | 16 |
| Muscular dystrophies | D009136 | HP_0003560 | G71.0 | — | 9 | 5 | — | 2 | 16 |
| Inborn genetic diseases | D030342 | EFO_0000508 | — | — | — | 3 | — | — | 3 |
| Muscular diseases | D009135 | HP_0003198 | G72.9 | — | — | 3 | — | — | 3 |
| Nervous system diseases | D009422 | — | G00-G99 | — | — | 3 | — | — | 3 |
| Musculoskeletal diseases | D009140 | — | — | — | — | 3 | — | — | 3 |
| X-linked genetic diseases | D040181 | — | — | — | — | 3 | — | — | 3 |
| Neuromuscular diseases | D009468 | EFO_1001902 | G70.9 | — | — | 3 | — | — | 3 |
| Atrophic muscular disorders | D020966 | — | — | — | — | 3 | — | — | 3 |
Indications Phases 2
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Aniridia | D015783 | HP_0000526 | Q13.1 | — | 2 | — | — | — | 2 |
| Hemophilia a | D006467 | EFO_0007267 | D66 | — | 1 | — | — | — | 1 |
| Hemophilia b | D002836 | — | D67 | — | 1 | — | — | — | 1 |
| Colorectal neoplasms | D015179 | — | — | 1 | 1 | — | — | — | 1 |
| Endometrial neoplasms | D016889 | EFO_0004230 | — | 1 | 1 | — | — | — | 1 |
| Epilepsy | D004827 | EFO_0000474 | G40.9 | — | 1 | — | — | — | 1 |
| Myoclonic epilepsies | D004831 | HP_0002123 | G40.4 | — | 1 | — | — | — | 1 |
| Inborn errors metabolism | D008661 | — | — | — | 1 | — | — | — | 1 |
| Inborn errors amino acid metabolism | D000592 | — | E72.9 | — | 1 | — | — | — | 1 |
Indications Phases 1
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Healthy volunteers/patients | — | — | — | 1 | — | — | — | — | 1 |
Indications Without Phase
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Epidemiology
Epidemiological information for investigational and approved indications
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Drug
General
| Drug common name | Ataluren |
| INN | ataluren |
| Description | 3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid is a ring assembly and an oxadiazole. |
| Classification | Small molecule |
| Drug class | inducers of ribosomal readthrough on nonsense mutation mRNA stop codons |
| Image (chem structure or protein) |  |
| Structure (InChI/SMILES or Protein Sequence) | O=C(O)c1cccc(-c2noc(-c3ccccc3F)n2)c1 |
Identifiers
| PDB | — |
| CAS-ID | 775304-57-9 |
| RxCUI | — |
| ChEMBL ID | CHEMBL256997 |
| ChEBI ID | — |
| PubChem CID | 11219835 |
| DrugBank | DB05016 |
| UNII ID | K16AME9I3V (ChemIDplus, GSRS) |
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Translarna – PTC Therapeutics
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Safety
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Adverse Events
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