Product Description
Ataluren is an orally delivered, investigational drug that has the potential to overcome the effects of the nonsense mutation. For Patients With Nonsense Mutation Dystrophinopathy (Sourced from: https://clinicaltrials.gov/ct2/show/NCT01826487)
Mechanisms of Action: Unknown
Novel Mechanism: No
Modality: Small Molecule
Route of Administration: Oral
FDA Designation: None *
Approval Status: Not Approved
Approved Countries: Austria | Belgium | Brazil | Chile | Croatia | Czech | Denmark | Estonia | European Medicines Agency | Finland | France | Germany | Hungary | Iceland | Ireland | Israel | Italy | Korea | Latvia | Lithuania | Netherlands | Norway | Peru | Poland | Portugal | Romania | Russia | Saudi Arabia | Slovakia | Slovenia | Spain | Sweden | Ukraine | United Kingdom
Approved Indications: None
Known Adverse Events: None
Company: PTC Therapeutics
Company Location: SOUTH PLAINFIELD NJ 07080-2449
Company CEO: Stuart W. Peltz
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic: Australia, Brazil, Bulgaria, Canada, China, Hong Kong, India, Japan, Korea, Malaysia, Mexico, Netherlands, Poland, Puerto Rico, Russia, Taiwan, Thailand, Turkey, United States
Active Clinical Trial Count: 11
Highest Development Phases
Phase 3: Genetic Diseases, X-Linked|Muscular Disorders, Atrophic|Muscular Dystrophy, Duchenne
Phase 2: Colorectal Cancer|Endometrial Cancer
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| jRCT2041200087 | P3 |
Recruiting |
Muscular Dystrophy, Duchenne |
2024-01-31 |
|
| nmDMD | P2 |
Completed |
Muscular Dystrophy, Duchenne |
2023-08-07 |
|
| ATAPEMBRO | P2 |
Recruiting |
Endometrial Cancer|Colorectal Cancer |
2023-06-01 |
|
| ATAPEMBRO | P2 |
Active, not recruiting |
Colorectal Cancer|Endometrial Cancer |
2023-02-20 |