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Golodirsen - Sarepta Therapeutics

Drug Profile

Golodirsen - Sarepta Therapeutics

Alternative Names: Exon 53 PMO; SRP 4053; Vyondys 53

Latest Information Update: 27 Oct 2023

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At a glance

  • Originator University of Western Australia
  • Developer Sarepta Therapeutics
  • Class Antisense oligonucleotides; Morpholines
  • Mechanism of Action Dystrophin expression stimulants; RNA interference
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Duchenne muscular dystrophy
  • New Molecular Entity 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)
  • 21 Aug 2023 Sarepta Therapeutics terminates a phase-III clinical trial in Duchenne muscular dystrophy (In adolescents, In children, In adults) in USA, United Kingdom, Belgium, Italy, Spain, Bulgaria, Canada, Czech Republic, France, Germany, Israel, Poland and Sweden (IV) due to the reason that participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment (NCT03532542) (EudraCT2017-004625-32)
  • 31 Dec 2022 Sarepta Therapeutics receives patent allowance for golodirsen, covering "Composition of Matter" in Europe (before December 2022)
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