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Viltolarsen - Nippon Shinyaku

Drug Profile

Viltolarsen - Nippon Shinyaku

Alternative Names: Biltrarsen; Exon 53 skipping antisense oligonucleotide; Exon 53 skipping DMD therapeutic; NCNP-01; NS 065; NS-065/NCNP-01; VILTEPSO

Latest Information Update: 12 Jun 2024

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At a glance

  • Originator National Center of Neurology and Psychiatry; Nippon Shinyaku
  • Developer National Center of Neurology and Psychiatry; Nippon Shinyaku; NS Pharma
  • 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

  • 13 Apr 2024 Updated efficacy data from the phase II Galactic53 trial in Duchenne muscular dystrophy presented at the 76th Annual Meeting of the American Academy of Neurology (AAN-2024)
  • 06 Mar 2024 Efficacy and adverse events data from phase II trial in Duchenne muscular dystrophy released by NS Pharma
  • 19 Oct 2023 NS Pharma completes the phase-III RACER53 trial in Duchenne muscular dystrophy (In children) in Australia, Greece, Italy, South Korea, Netherlands, New Zealand, Norway, Russia, Spain, Taiwan, Turkey, Canada, Sweden, Chile, China, Japan, Hong Kong, Mexico, Ukraine, United Kingdom and USA (NCT04060199)
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