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About this data Sourced primarily from ClinicalTrials.gov — a listing isn't proof of safety or FDA approval.

Pipeline and clinical trial information here comes primarily from ClinicalTrials.gov. A listing on ClinicalTrials.gov means a study has been registered — not that a treatment is proven safe, effective, or FDA-approved. Investigational treatments may never be approved. Who can join a trial is decided only by that trial's own study team.

Not yet reported

EPI-743 200 mg

Company
PTC Therapeutics
Mechanism
Mechanism not yet reported
Route
Route not yet reported
Recruiting trials
None currently listed

Efficacy results: Not yet reported

No recruiting studies currently listed

See all studies for this drug

Data source: ClinicalTrials.gov · trial data as of Oct 1, 2026

Trial details
  • Completed NCT NCT01728064

    Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia

    Not yet reported PTC Therapeutics 60 participants Est. completion 2016-02-29 3 sites
    Other conditions: Friedreich's Ataxia
    View official trial listing →
  • Completed NCT NCT02257983

    Protective Effects of EPI-743 on Noise-Induced Hearing Loss

    Not yet reported Edison Pharmaceuticals Inc 77 participants Est. completion 2016-02-29 1 site
    Other conditions: Noise-induced Hearing Loss
    View official trial listing →
Efficacy history

Not yet reported.

Safety

Not yet reported.

Sources

Also known as: EPI-743 200 mg, EPI-743 400 mg

Related On The Pen coverage

No related coverage yet.

This drug may be investigational and not yet approved for any use. Whether someone can join a clinical trial is determined only by that trial's own study team — On The Pen does not determine eligibility. Information here can change; always confirm current details on the official ClinicalTrials.gov listing or with the sponsor.