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Ataluren (PTC124®)
Ataluren (PTC124®) is a Nonsense suppression agent Small molecule drug developed by PTC Therapeutics. It is currently in Phase 3 development for Duchenne muscular dystrophy (DMD) with nonsense mutations, Cystic fibrosis with nonsense mutations, Hemophilia B Leyden.
Ataluren allows ribosomes to read through premature stop codons (nonsense mutations) and produce full-length, functional proteins.
Ataluren allows ribosomes to read through premature stop codons (nonsense mutations) and produce full-length, functional proteins. Used for Duchenne muscular dystrophy (DMD) with nonsense mutations, Cystic fibrosis with nonsense mutations, Hemophilia B Leyden.
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Baseline phase 3 → approval rate
+58.3pp
Industry-wide phase 3 drugs reach approval ~58.3% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas). -
Rare-disease pathway favourability
+5.0pp
Rare-disease drugs benefit from FDA Orphan Drug Act, smaller pivotal trials, and more flexible endpoints. Approval rates run ~5pp above baseline.
| Regulator | Country | Likely year | Lag vs FDA |
|---|---|---|---|
| FDA | US | 2028–2030 | — |
| EMA | EU | 2029–2031 | +0.7 yr |
| MHRA | GB | 2029–2031 | +0.7 yr |
| Health Canada | CA | 2029–2032 | +0.9 yr |
| TGA | AU | 2029–2032 | +1.2 yr |
| PMDA | JP | 2029–2032 | +1.5 yr |
| NMPA | CN | 2030–2033 | +2.3 yr |
| MFDS | KR | 2029–2032 | +1.4 yr |
| CDSCO | IN | 2029–2033 | +1.8 yr |
| ANVISA | BR | 2030–2033 | +2.3 yr |
Hover any row for the lag rationale. Lag estimates are reduced when the drug has FDA Breakthrough or EMA PRIME designation (sponsors file globally in parallel).
Estimate based on the BIO/Informa industry phase transition rates plus per-drug modifiers for therapeutic area, sponsor type, FDA designations, mechanism, and trial design. Per-jurisdiction lags from Tufts CSDD international approval studies. Not investment, clinical or regulatory advice. Methodology: /methodology#likelihood.
At a glance
| Generic name | Ataluren (PTC124®) |
|---|---|
| Sponsor | PTC Therapeutics |
| Drug class | Nonsense suppression agent |
| Target | Ribosome (eukaryotic translation machinery) |
| Modality | Small molecule |
| Therapeutic area | Rare genetic diseases |
| Phase | Phase 3 |
Mechanism of action
Ataluren is a small-molecule nonsense suppression agent that binds to the ribosome and enables translational read-through of premature termination codons (PTCs) caused by nonsense mutations. By allowing the ribosome to insert an amino acid at the stop codon position instead of terminating translation, the drug permits synthesis of full-length, partially functional protein. This mechanism is particularly relevant for genetic diseases caused by nonsense mutations, where the underlying defect is a single point mutation creating a premature stop signal.
Approved indications
- Duchenne muscular dystrophy (DMD) with nonsense mutations
- Cystic fibrosis with nonsense mutations
- Hemophilia B Leyden
Common side effects
- Headache
- Diarrhea
- Nausea
- Upper respiratory tract infection
Key clinical trials
- Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada (PHASE3)
- Extension Study of Ataluren (PTC124) in Cystic Fibrosis (PHASE3)
- Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy (PHASE2)
- Ataluren for Nonsense Mutation Methylmalonic Acidemia (PHASE2)
- Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF) (PHASE3)
- Effects of Rifampin on the Pharmacokinetics of Ataluren (PHASE1)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Ataluren (PTC124®) CI brief — competitive landscape report
- Ataluren (PTC124®) updates RSS · CI watch RSS
- PTC Therapeutics portfolio CI
Frequently asked questions about Ataluren (PTC124®)
What is Ataluren (PTC124®)?
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What drug class is Ataluren (PTC124®) in?
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What does Ataluren (PTC124®) target?
Related
- Drug class: All Nonsense suppression agent drugs
- Target: All drugs targeting Ribosome (eukaryotic translation machinery)
- Manufacturer: PTC Therapeutics — full pipeline
- Therapeutic area: All drugs in Rare genetic diseases
- Indication: Drugs for Duchenne muscular dystrophy (DMD) with nonsense mutations
- Indication: Drugs for Cystic fibrosis with nonsense mutations
- Indication: Drugs for Hemophilia B Leyden
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing