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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
Therapeutic Area
MeSH
musculoskeletal diseasesD009140
nervous system diseasesD009422
hereditary congenital and neonatal diseases and abnormalitiesD009358
Trade Name
FDA
EMA
No data
Drug Products
FDA
EMA
New Drug Application (NDA)
New Drug Application (NDA)
Abbreviated New Drug Application (ANDA)
Abbreviated New Drug Application (ANDA)
No data
Labels
FDA
EMA
No data
Indications
FDA
EMA
No data
Agency Specific
FDA
EMA
No data
Patent Expiration
No data
ATC Codes
M: Musculo-skeletal system drugs
— M09: Other drugs for disorders of the musculo-skeletal system in atc
— M09A: Other drugs for disorders of the musculo-skeletal system in atc
— M09AX: Other drugs for disorders of the musculo-skeletal system in atc
— M09AX03: Ataluren
HCPCS
No data
Clinical
Clinical Trials
32 clinical trials
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Indications Phases 4
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
Cystic fibrosisD003550EFO_0000390E84—452—11
Indications Phases 3
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
Duchenne muscular dystrophyD020388—85——13
Muscular dystrophiesD009136EFO_0000757G71.0—13——4
Inborn genetic diseasesD030342EFO_0000508——3——3
Muscular diseasesD009135EFO_0002970G72.9——3——3
Nervous system diseasesD009422G00-G99——3——3
Musculoskeletal diseasesD009140——3——3
X-linked genetic diseasesD040181——3——3
Neuromuscular diseasesD009468EFO_1001902G70.9——3——3
Atrophic muscular disordersD020966——3——3
Indications Phases 2
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
AniridiaD015783EFO_0004132Q13.1—2———2
Hemophilia aD006467EFO_0007267D66—1———1
Hemophilia bD002836D67—1———1
Colorectal neoplasmsD01517911———1
Endometrial neoplasmsD016889EFO_000423011———1
EpilepsyD004827EFO_0000474G40.9—1———1
Inborn errors amino acid metabolismD000592E72.9—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
No data
Epidemiology
Epidemiological information for investigational and approved indications
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Drug
General
Drug common nameATALUREN
INNataluren
Description
3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid is a ring assembly and an oxadiazole.
Classification
Small molecule
Drug classinducers 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-ID775304-57-9
RxCUI—
ChEMBL IDCHEMBL256997
ChEBI ID—
PubChem CID11219835
DrugBankDB05016
UNII IDK16AME9I3V (ChemIDplus, GSRS)
Target
Agency Approved
No data
Alternate
No data
Variants
Clinical Variant
No data
Financial
Translarna - PTC Therapeutics
$
€
£
₣
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Tabular view
Trends
PubMed Central
Top Terms for Disease or Syndrome:
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Additional graphs summarizing 1,197 documents
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Safety
Black-box Warning
No Black-box warning
Adverse Events
Top Adverse Reactions
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4,706 adverse events reported
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