Back to discover
Drug

Ataluren

Regulatory activity · EMA
Emerging researchLate-stage development
RxNorm1030912ChEMBLCHEMBL256997UNIIK16AME9I3V
2
Research papers
1
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

A Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension

Clinical trial2026-03-10Results posted · ClinicalTrials.gov

An Open-Label, Safety Study for Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy

Clinical trial2026-02-10Completed · ClinicalTrials.gov

Market withdrawal: Translarna (EMA)

Regulatory2025-03-28EMA

An Open-Label Study Evaluating the Safety and Pharmacokinetics of Ataluren in Children From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy

Clinical trial2024-04-01Results posted · ClinicalTrials.gov

A Phase 2 Randomized, Double-Masked Placebo-Controlled Crossover Safety and Tolerability Study of Ataluren for Drug Resistant Epilepsy in Patients With Nonsense Mutation CDKL5 or Dravet Syndrome

Clinical trial2023-02-16Results posted · ClinicalTrials.gov

A Phase 2, Multicenter, Randomized, Double-Masked, Placebo-Controlled Study of the Safety and Efficacy of Ataluren (PTC124) for the Treatment of Nonsense Mutation Aniridia

Clinical trial2022-05-27Results posted · ClinicalTrials.gov

Phase 2 Clinical Pharmacology Study to Assess Dystrophin Levels in Subjects With nmDMD Before and After Treatment With Ataluren

Clinical trial2022-04-05Results posted · ClinicalTrials.gov

Phase 2, Non-Interventional, Clinical Study to Assess Dystrophin Levels in Subjects With Nonsense Mutation Duchenne Muscular Dystrophy Who Have Been Treated With Ataluren for ≥9 Months

Clinical trial2022-04-05Results posted · ClinicalTrials.gov

Ataluren for drug-resistant epilepsy in nonsense variant-mediated Dravet syndrome and CDKL5 deficiency disorder.

Research2021-02-04Annals of clinical and translational neurology

Nonsense Mutation Aniridia: An Ataluren (PTC124) Phase 2 Extension Study

Clinical trial2021-01-31Withdrawn · ClinicalTrials.gov

An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients With Nonsense Mutation Dystrophinopathy

Clinical trial2020-11-25Results posted · ClinicalTrials.gov

Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial.

Research2014-05-15The Lancet. Respiratory medicine

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Ataluren
RxNorm CUI
1030912
ChEMBL ID
CHEMBL256997
UNII
K16AME9I3V
Regulatory jurisdictions
ema

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Authorities
EMA
Total events
1
emaEuropean Medicines Agency· 1 event
2025-03-28Market withdrawalMarket
Market withdrawal: Translarna (EMA)
Indication: Translarna is indicated for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years and older.… Show full indication

Translarna is indicated for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years and older. Efficacy has not been demonstrated in non-ambulatory patients. The presence of a nonsense mutation in the dystrophin gene should be determined by genetic testing.

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

27 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
27
registered trials across all phases
LATEST COMPLETION 2026
11
Late-stage (III+)
18
Completed
9
Discontinued
PHASE DISTRIBUTIONn = 27
Phase 11Phase 215Phase 310Phase 41

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

An Open Label N of 1 Study to Evaluate the Study and Efficacy of Long-Term Treatment With Ivacaftor in Combination With Ataluren (PTC124) in Subjects With Nonsense Mutation Cystic Fibrosis

CompletedPH IVUniversity of Alabama at Birminghamn=12018

An Open-Label, Safety Study for Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy

CompletedPH IIIPTC Therapeuticsn=2702026

A Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension

CompletedPH IIIPTC Therapeuticsn=3602023

A Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy

TerminatedPH IIIPTC Therapeuticsn=2192018

An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients With Nonsense Mutation Dystrophinopathy

CompletedPH IIIPTC Therapeuticsn=942018

Recent completions

Trials that read out recently, adding to the completed evidence base.

An Open-Label, Safety Study for Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy

CompletedPH IIIPTC Therapeuticsn=2702026

An Open-Label Study Evaluating the Safety and Pharmacokinetics of Ataluren in Children From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy

CompletedPH IIPTC Therapeuticsn=62023

A Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension

CompletedPH IIIPTC Therapeuticsn=3602023

A Phase 2 Randomized, Double-Masked Placebo-Controlled Crossover Safety and Tolerability Study of Ataluren for Drug Resistant Epilepsy in Patients With Nonsense Mutation CDKL5 or Dravet Syndrome

CompletedPH IINYU Langone Healthn=152021

A Phase 2, Multicenter, Randomized, Double-Masked, Placebo-Controlled Study of the Safety and Efficacy of Ataluren (PTC124) for the Treatment of Nonsense Mutation Aniridia

CompletedPH IIPTC Therapeuticsn=392021

Phase 2 Clinical Pharmacology Study to Assess Dystrophin Levels in Subjects With nmDMD Before and After Treatment With Ataluren

CompletedPH IIPTC Therapeuticsn=202020

Research activity

2 papers

Key research shaping understanding of this drug, combining the latest publications with the most influential evidence.

Publications over time
20142021
Journals, researchers & institutions
Top journals
  • Annals of clinical and translational neurology1
  • The Lancet. Respiratory medicine1
Leading researchers
  • Accurso FJ1
  • Ajayi T1
  • Barth J1
  • Bluvstein J1
  • Branstrom A1
  • Bronsveld I1
  • De Boeck K1
  • Devinsky O1
Leading institutions
free-text, unnormalised
  • Ann & Robert H Lurie Children's Hospital of Chicago1
  • Center for Cystic Fibrosis1
  • Centre de Ressource et de Competence de la Mucoviscidose1
  • Centre for Infection and Immunity1
  • Children's Hospital Colorado1
  • Cystic Fibrosis Centre1
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
  • Europe PMC — research literature
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.