Drug Profile
Eteplirsen - Sarepta Therapeutics
Alternative Names: AVI 4658; AVI-4658 PMO; Exondys 51Latest Information Update: 05 Nov 2023
Price :
$50
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At a glance
- Originator University of Western Australia
- Developer Sarepta Therapeutics
- Class Antisense oligonucleotides; Morpholines
- Mechanism of Action Dystrophin expression modulators; RNA interference
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity Yes
- Available For Licensing Yes
Highest Development Phases
- Marketed Duchenne muscular dystrophy
Most Recent Events
- 01 Sep 2023 Nationwide Children's Hospital completes phase II trial in Duchenne muscular dystrophy (In infants, In adolescents, In children, In adults) in USA (IV) (NCT04179409)
- 31 Dec 2022 Sarepta Therapeutics receives patent allowance for eteplirsen, covering "composition of matter and methods of use", "method of use" and "composition of matter" in USA and Europe (before December 2022)
- 29 Sep 2022 Sarepta Therapeutics terminates a phase II trial due to patients were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment, for Duchenne muscular dystrophy (Treatment-experienced) in United Kingdom, Belgium, France and Italy (IV) (NCT03985878)