Eteplirsen
- Generic Name
- Eteplirsen
- Brand Names
- Exondys
- Drug Type
- Biotech
- CAS Number
- 1173755-55-9
- Unique Ingredient Identifier
- AIW6036FAS
- Background
Eteplirsen is a synthetic antisense oligonucleotide and a phosphorodiamidate morpholino oligomer. It consists of a six-membered morpholino ring replacing the five-membered ribofuranosyl rings found in natural DNA and RNA.
Duchenne muscular dystrophy is a rare genetic disorder characterized by progressive muscle deterioration and premature death most commonly due to respiratory or cardiac complications. It is caused by loss-of-function mutations in the DMD gene coding for dystrophin, an essential protein involved in maintaining the structural integrity and function of muscle fibres. Eteplirsen was first approved by the FDA in September 2016 for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the DMD gene, which codes for dystrophin, that is amenable to exon 51 skipping. Eteplirsen directly works on the DMD gene to promote dystrophin production. Eteplirsen was the first treatment for DMD approved by the FDA.
- Indication
Eteplirsen is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with eteplirsen.
- Associated Conditions
- Duchenne Muscular Dystrophy (DMD)
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- First Posted Date
- 2024-09-23
- Last Posted Date
- 2024-09-23
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- Target Recruit Count
- 300
- Registration Number
- NCT06606340
- Locations
- 🇺🇸
Arkansas Children's Hospital Research Institute, Little Rock, Arkansas, United States
🇺🇸Children's Hospital Los Angeles, Los Angeles, California, United States
🇺🇸University of California Davis Medical Center, Sacramento, California, United States
A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications.
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- First Posted Date
- 2019-11-27
- Last Posted Date
- 2023-10-26
- Lead Sponsor
- Kevin Flanigan
- Target Recruit Count
- 3
- Registration Number
- NCT04179409
- Locations
- 🇺🇸
Nationwide Children's Hospital, Columbus, Ohio, United States
A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)
- First Posted Date
- 2019-06-20
- Last Posted Date
- 2024-12-20
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- Target Recruit Count
- 160
- Registration Number
- NCT03992430
- Locations
- 🇮🇳
Royal Institute of Child Neurosciences, Ahmedabad, India
🇩🇪Charité Universitätsmedizin Berlin CVK, Berlin, Germany
🇮🇳Aster RV Hospital, Bengaluru, India
A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995)
- First Posted Date
- 2019-06-14
- Last Posted Date
- 2023-08-18
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- Target Recruit Count
- 15
- Registration Number
- NCT03985878
- Locations
- 🇫🇷
Hopital Trousseau, Bâtiment lemariey, Paris, France
🇧🇪UZ-Gent, Gent, Belgium
🇮🇹Fondazione Policlinico Universitario Agostino Gemelli, UOC Neuropsichiatria Infantile, Rome, Italy
Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 Skipping
- First Posted Date
- 2017-07-17
- Last Posted Date
- 2021-12-09
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- Target Recruit Count
- 15
- Registration Number
- NCT03218995
- Locations
- 🇧🇪
Universitair ziekenhuis Gent, Gent, Belgium
🇫🇷Armand-Trousseau Hospital, Paris, France
🇮🇹Site Fondazione Policlinico Universitario Agostino Gemelli, Roma, Italy
Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy
- First Posted Date
- 2015-04-17
- Last Posted Date
- 2021-01-25
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- Target Recruit Count
- 33
- Registration Number
- NCT02420379
- Locations
- 🇺🇸
University of Iowa Children's Hospital, Iowa City, Iowa, United States
🇺🇸Rare Disease Research Center, Atlanta, Georgia, United States
🇺🇸Children's Hospital of Atlanta, Atlanta, Georgia, United States
Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy
- First Posted Date
- 2014-11-10
- Last Posted Date
- 2020-03-30
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- Target Recruit Count
- 24
- Registration Number
- NCT02286947
- Locations
- 🇺🇸
Massachusetts General Hospital, Boston, Massachusetts, United States
🇺🇸University of Rochester Medical Center, Rochester, New York, United States
🇺🇸Nationwide Children's Hospital, Columbus, Ohio, United States
Study of Eteplirsen in DMD Patients
- First Posted Date
- 2014-10-02
- Last Posted Date
- 2021-01-25
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- Target Recruit Count
- 109
- Registration Number
- NCT02255552
- Locations
- 🇺🇸
Penn State Hershey Medical Center, Hershey, Pennsylvania, United States
🇺🇸David Geffen School of Medicine at UCLA, Los Angeles, California, United States
🇺🇸Emory University, Atlanta, Georgia, United States
Drug Development Updates
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