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Ataluren

Alternative Names: ataluren, ptc124, translarna
Latest Update: 2024-11-06
Latest Update Note: News Article

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

Map of Global Clinical Trials for Ataluren

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

Endometrial Cancer|Colorectal Cancer

2023-02-20

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