Translarna
(Ataluren)Phase 3Small molecule
Ataluren is used to treat a rare genetic muscle disease called Duchenne muscular dystrophy (DMD) that is caused by a specific type of genetic mutation. It works by allowing the body to produce functional muscle protein despite the genetic defect.[1] Translarna is currently being investigated across 11 indications[3], with its most advanced program in Phase 3.[1]
Translarna is one of 210 small molecule programs targeting Muscular Dystrophy. It is currently in Phase 3.
11
Indications
27
Total Trials
0
Active Trials
209
Competitors
Pipeline
Status by Indication[3][1]
Indication
Showing 10 of 11 indications
Competition
Competitive Landscape
386 competitors in Cystic Fibrosis
#DrugCompanyPhaseTrials
1Translarna
PPTC Therapeutics
Phase 3
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UUniversity of Aarhus
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Advil
KKenvue Brands
Preclinical
Tums
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Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.
Clinical
Trials by Indication[1]
Duchenne Muscular Dystrophy7 trials
Show 4 more trials
Cystic Fibrosis7 trials
Becker muscular dystrophy4 trials
ART2 trials
5 more indications
Unspecified1 trial
Sources
References
- [1]
- [2]
- [3]Open Targets Platform — Drug–disease associations for Ataluren. EMBL-EBI & Wellcome Sanger Institute.
- [4]
Details
Drug Info
Quick Facts
- Mechanism of Action
- 80s Ribosome Modulator[2]
- Modality
- Small molecule[2]
- Highest Phase
- Phase 3[1]
- Company
- PPTC Therapeutics