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Casimersen - Sarepta Therapeutics

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Drug Profile

Casimersen - Sarepta Therapeutics

Alternative Names: AMONDYS 45; SRP-4045

Latest Information Update: 05 Nov 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 casimersen, covering "composition of matter and methods of use" in USA (before December 2022)

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