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; VILTEPSOLatest Information Update: 12 Mar 2024
Price :
$50
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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
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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
Highest Development Phases
- Marketed Duchenne muscular dystrophy
Most Recent Events
- 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)
- 13 Jul 2023 NS Pharma and Nippon Shinyaku complete the phase II Galactic53 trial in Duchenne muscular dystrophy (In children, In adolescents, In adults, In the elderly) in USA, China, Italy, Russia, Spain, Turkey (IV, Infusion) (NCT04956289)