Drug

Ataluren

Withdrawn from market

Class80S Ribosome modulatorResearchRetrospective safety research

RxNorm1030912 ChEMBLCHEMBL256997 ATCM09AX03UNIIK16AME9I3V

Research papers

Protein targets

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

ATC codes

M09AX03

UNII

K16AME9I3V

Primary mechanism

80S Ribosome modulator

Regulatory jurisdictions

ema

Pharmacology & targets

78 targets

Known molecular targets and mechanisms supported by curated pharmacology databases.

Small ribosomal subunit protein uS2

RPSA · P08865