Ataluren 125mg granules for oral suspension sachets
Requires a prescription from a doctor or prescriber
Ataluren is a novel, orally administered drug that targets nonsense mutations.
Official documents, adverse reaction reporting, and safety monitoring
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Official medicine documents
Yellow Card
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Drug safety updates
MHRA alerts for Ataluren
Safety monitoring data
Yellow Card reports
The MHRA Yellow Card scheme collects reports of suspected side effects from healthcare professionals and patients. View the Drug Analysis Profile (iDAP) for real-world adverse reaction data.
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Suspected adverse reactions reported for Ataluren
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Report a side effect
Submit a Yellow Card report to the MHRA
Data from the MHRA Yellow Card scheme. A reported reaction does not necessarily mean the medicine caused it. Contains public sector information licensed under the Open Government Licence v3.0.
EudraVigilance
The European Medicines Agency (EMA) collects suspected adverse reaction reports from across the EU/EEA through the EudraVigilance system. Search for safety data on this medicine.
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Suspected adverse reactions reported for Ataluren
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EudraVigilance data is published by the European Medicines Agency (EMA). A suspected adverse reaction is not necessarily caused by the medicine.
1 branded products available
MHRA licensed products
View all licensed products for Ataluren on the MHRA register
Translarna 125mg granules for oral suspension sachets
Therapeutically similar medicines
Similarity is based on WHO Anatomical Therapeutic Chemical (ATC) classification and on a factual NHS dm+d therapeutic-grouping code prefix. Source data: NHS dm+d via TRUD (OGL v3.0), WHO ATC/DDD Index.
NHS prescribing volume and spending trends
Guidelines from the National Institute for Health and Care Excellence
NICE clinical guidance(5)
Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene (HST22)
Mexiletine for treating the symptoms of myotonia in non-dystrophic myotonic disorders (TA748)
Asfotase alfa for treating paediatric-onset hypophosphatasia (HST23)
Vamorolone for treating Duchenne muscular dystrophy in people 4 years and over (TA1031)
Givinostat for treating Duchenne muscular dystrophy in people 6 years and over (TA1157)
Source: National Institute for Health and Care Excellence (NICE). Contains public sector information licensed under the Open Government Licence v3.0.
Check stock at pharmacies and supply information
Pharmacy stock checkers
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Supply & safety information
Official UK regulator monitoring and safety alerts
Pharmacy links redirect to the retailer's own search and do not represent real-time stock levels. Shortage and safety information sourced from MHRA drug safety updates (gov.uk, Crown Copyright under OGL v3.0).
Codes for healthcare professionals and prescribing systems
These codes are used by healthcare IT systems and prescribers to identify this medicine.
NHS UK identifiers
Browse tools
SNOMED CT and dm+d codes from NHS TRUD (Technology Reference data Update Distribution), licensed under the Open Government Licence v3.0. ATC codes from the WHO Collaborating Centre for Drug Statistics Methodology (whocc.no).
Active and completed clinical studies from ClinicalTrials.gov
Source: ClinicalTrials.gov, a database of the U.S. National Library of Medicine (NLM), National Institutes of Health (NIH). Data accessed via ClinicalTrials.gov API v2. Trial information is provided for research purposes and does not constitute medical advice.
Academic studies and reviews for this medicine's active substance
Showing the 50 most relevant studies.
Reviews & meta-analyses: 10 · Randomised trials: 12 · 2010–2026
Showing the 50 most relevant studies, sorted by most relevant.
Craig M. McDonald, C. Campbell, R. Torricelli, et al.
Lancet, 2017
E. Kerem, M. Konstan, K. De Boeck, et al.
The Lancet. Respiratory medicine, 2014
Gabriela Carolino, Amanda Rabelo, Carolina Alves Côrrea, et al.
Neurology, 2026
Yuh-Jyh Jong, P. Karachunski, J. Statland, et al.
Neurology, 2024
Bijoyita Roy, W. Friesen, Y. Tomizawa, et al.
Proceedings of the National Academy of Sciences, 2016
M. Konstan, Emily DiMango, E. Kerem, et al.
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2017
Noreen Zainal Abidin, Iram J. Haq, Aaron I. Gardner, et al.
Expert Opinion on Pharmacotherapy, 2017
C. Campbell, R. Barohn, E. Bertini, et al.
Journal of comparative effectiveness research, 2020
N Goemans, C Campbell, CM McDonald, et al.
Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques, 2016
Manuel Haas, Viktor Vlček, Pavel Balabanov, et al.
Neuromuscular disorders : NMD, 2015
Sources: aggregated from Europe PMC (EMBL-EBI), OpenAlex, Crossref, PubMed and other open scholarly databases. Retracted articles are excluded. Study information is provided for research purposes and does not constitute medical advice.
Pharmacology and chemical data from DrugBank
Key facts
Drug status
Approved
Major interactions
None known
Half-life
2-6 hours
Mechanism
Ataluren enables ribosomal readthrough of mRNA containing premature stop codons…
Food interactions
None known
Human targets
1 target
Data: DrugBank · CC BY-NC 4.0
Pharmacokinetics at a glance
Absorption
1.5 hours
[L925]
…
Half-life
2-6 hours
[L925]
Protein binding
99.6%
Metabolism
8%
Elimination
1%
Pharmacokinetic data: DrugBank · CC BY-NC 4.0
This drug does not yet have approval by the US Food and Drug Administration or by Health Canada for any indications.
Known interactions with other medicines. Always consult a healthcare professional.
Showing 50 of 116 interactions
The research on the effects of Ataluren on the translation and stability of nonsense-containing mRNA in vitor show that Ataluren promoted readthrough at each of the nonsense codons, showing maximal activity with UGA, while having no effect on mRNA levels. Unlike the stable cell line assays, Ataluren did not discriminate significantly between the UAG and UAA mRNAs. Ataluren was a more potent nonsense-suppressing agent than gentamicin, and exhibited 4- to 15-fold stimulation of in vitro readthrough relative to the controls at levels similar to those in the stable cell reporter assays. These results indicate that Ataluren modulates termination efficiency at premature nonsense codons.
How the body processes this drug — absorption, distribution, metabolism, and elimination
[L925]
[L925]
[L925]
[L925]
[L925]
Proteins and enzymes this drug interacts with in the body
Also implicated in signaling events and synaptic transmission
Enzymes involved in drug metabolism — important for understanding drug interactions
ATC M09AX03
Chemical identifiers
CAS, UNII, InChI Key and database cross-references
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Chemical identifiers
CAS, UNII, InChI Key and database cross-references
Linked compound data from DrugBank Open Data (CC BY-NC 4.0)
Ataluren
Additional database identifiers
ChemSpider
9394889
PDB
JBF
ZINC
ZINC000013831791
HUGO Gene Nomenclature Committee (HGNC)
HGNC:2928
GeneCards
DMD
UniProt Accession
DMD_HUMAN
HUGO Gene Nomenclature Committee (HGNC)
HGNC:12541
GeneCards
UGT1A9
GenBank Gene Database
S55985
GenBank Protein Database
7690346
UniProt Accession
UD19_HUMAN
HUGO Gene Nomenclature Committee (HGNC)
HGNC:10970
GenAtlas
hROAT1
GeneCards
SLC22A6
GenBank Gene Database
AF057039
GenBank Protein Database
3831566
Guide to Pharmacology
1025
UniProt Accession
S22A6_HUMAN
HUGO Gene Nomenclature Committee (HGNC)
HGNC:10972
GeneCards
SLC22A8
GenBank Gene Database
AF097491
GenBank Protein Database
4378059
Guide to Pharmacology
1027
UniProt Accession
S22A8_HUMAN
HUGO Gene Nomenclature Committee (HGNC)
HGNC:10961
GeneCards
SLCO1B3
GenBank Gene Database
AJ251506
GenBank Protein Database
9187497
Guide to Pharmacology
1221
UniProt Accession
SO1B3_HUMAN
DrugBank citations
If you use DrugBank data in your research, please cite:
- DrugBank 6.02024Recommended citationKnox C., Wilson M., Klinger C.M., et alDrugBank 6.0: the DrugBank Knowledgebase for 2024Nucleic Acids Res. 2024 Jan 552(D1):D1265-D1275
- DrugBank 5.02018Wishart D.S., Feunang Y.D., Guo A.C., et alDrugBank 5.0: a major update to the DrugBank database for 2018Nucleic Acids Res. 2017 Nov 846(D1):D1074-D1082
- DrugBank 4.02014Law V., Knox C., Djoumbou Y., et alDrugBank 4.0: shedding new light on drug metabolismNucleic Acids Res. 2014 Jan 142(1):D1091-7
- DrugBank 3.02011Knox C., Law V., Jewison T., et alDrugBank 3.0: a comprehensive resource for 'omics' research on drugsNucleic Acids Res. 2011 Jan39(Database issue):D1035-41
- DrugBank 2.02008Wishart D.S., Knox C., Guo A.C., et alDrugBank: a knowledgebase for drugs, drug actions and drug targets.Nucleic Acids Research2008 Jan36(Database issue):D901-6
- DrugBank 1.02006Wishart D.S., Knox C., Guo A.C., et alDrugBank: a comprehensive resource for in silico drug discovery and exploration.Nucleic Acids Research2006 Jan 134(Database issue):D668-72