Ataluren
- Generic Name
- Ataluren
- Brand Names
- Translarna
- Drug Type
- Small Molecule
- Chemical Formula
- C15H9FN2O3
- CAS Number
- 775304-57-9
- Unique Ingredient Identifier
- K16AME9I3V
- Background
Ataluren is a novel, orally administered drug that targets nonsense mutations. Ataluren is approved for use by the European Medicines Agency to treat Duchenne Muscular Dystrophy in patients aged 5 years and older who are able to walk. More specifically, ataluren is used in the small group of patients whose disease is caused by a specific genetic defect (called a ‘nonsense mutation’) in the dystrophin gene.
This drug does not yet have approval by the US Food and Drug Administration or by Health Canada for any indications.
- Indication
Ataluren is approved for use by the European Medicines Agency to treat Duchenne Muscular Dystrophy in patients aged 5 years and older who are able to walk. More specifically, ataluren is used in the small group of patients whose disease is caused by a specific genetic defect (called a ‘nonsense mutation’) in the dystrophin gene.
- Associated Conditions
- Duchenne Muscular Dystrophy (DMD)
- Associated Therapies
- -
A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
- First Posted Date
- 2020-04-07
- Last Posted Date
- 2024-04-01
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 6
- Registration Number
- NCT04336826
- Locations
- 🇺🇸
Rare Disease Research, LLC, Atlanta, Georgia, United States
A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia
- First Posted Date
- 2019-10-07
- Last Posted Date
- 2019-10-07
- Lead Sponsor
- PTC Therapeutics
- Registration Number
- NCT04117880
A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren
- First Posted Date
- 2019-01-08
- Last Posted Date
- 2022-04-05
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 6
- Registration Number
- NCT03796637
- Locations
- 🇺🇸
University of California, Los Angeles (UCLA), Los Angeles, California, United States
A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
- First Posted Date
- 2018-08-27
- Last Posted Date
- 2022-04-05
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 20
- Registration Number
- NCT03648827
- Locations
- 🇺🇸
University of Minnesota, Minneapolis, Minnesota, United States
🇺🇸Texas Children's Hospital, Houston, Texas, United States
🇺🇸University of California, Los Angeles (UCLA), Los Angeles, California, United States
PTC Study to Evaluate Ataluren in Combination With Ivacaftor
- First Posted Date
- 2017-08-22
- Last Posted Date
- 2020-03-26
- Lead Sponsor
- University of Alabama at Birmingham
- Target Recruit Count
- 1
- Registration Number
- NCT03256968
- Locations
- 🇺🇸
University of Alabama at Birmingham, Birmingham, Alabama, United States
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
- First Posted Date
- 2017-06-07
- Last Posted Date
- 2024-01-18
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 360
- Registration Number
- NCT03179631
- Locations
- 🇺🇸
Stanford University Medical Center, Palo Alto, California, United States
🇦🇺Queensland Children's Hospital, South Brisbane, Australia
🇺🇸Children's Hospital of Los Angeles, Los Angeles, California, United States
Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy
- First Posted Date
- 2016-06-30
- Last Posted Date
- 2020-08-28
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 14
- Registration Number
- NCT02819557
- Locations
- 🇺🇸
Children's Medical Center Dallas, Dallas, Texas, United States
🇺🇸University of Utah, Salt Lake City, Utah, United States
🇺🇸Rush University Medical Center, Chicago, Illinois, United States
Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome
- First Posted Date
- 2016-05-02
- Last Posted Date
- 2023-02-16
- Lead Sponsor
- NYU Langone Health
- Target Recruit Count
- 15
- Registration Number
- NCT02758626
- Locations
- 🇺🇸
New York University School of Medicine, New York, New York, United States
Study of Ataluren in Participants With Nonsense Mutation Aniridia
- First Posted Date
- 2016-01-06
- Last Posted Date
- 2022-05-27
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 39
- Registration Number
- NCT02647359
- Locations
- 🇺🇸
University of Virginia, Charlottesville, Virginia, United States
🇨🇦University of British Columbia, Vancouver, British Columbia, Canada
🇺🇸Casey Eye Institute, Oregon Health & Science University, Portland, Oregon, United States
Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis
- First Posted Date
- 2015-05-28
- Last Posted Date
- 2020-04-27
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 246
- Registration Number
- NCT02456103
- Locations
- 🇺🇸
University of Alabama at Birmingham, Birmingham, Alabama, United States
🇺🇸Miller Children's Hospital Long Beach, Long Beach, California, United States
🇺🇸Children's Hospital and Research Center at Oakland, Oakland, California, United States
Drug Development Updates
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