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Synonyms: NCNP-01 | NS-065 | Viltepso®
viltolarsen is an approved drug (FDA (2020))
Compound class:
Nucleic acid
Comment: Viltolarsen is an antisense phosphorodiamidate morpholino oligomer (PMO) class nucleotide drug. Its abbreviated chemical/nucleotide structure is all-P-ambo-[2',3'-azanediyl-P,2',3'-trideoxy-P-(dimethylamino)-2',3'-seco](2'-N→5')(CCTCCGGTTCTGAAGGTGTTC). Viltolarsen restores the reading frame of certain dystrophin gene mutations and promotes synthesis of a shorter but functional dystrophin protein.
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No information available. |
Summary of Clinical Use ![]() |
Used in the treatment of Duchenne muscular dystrophy (DMD), in patients with mutations amenable to exon 53 skipping [1]. In the EU, viltolarsen holds EMA orphan designation (granted in June 2020) for this indication. |
Clinical Trials | |||||
Clinical Trial ID | Title | Type | Source | Comment | References |
NCT04060199 | Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53) | Phase 3 Interventional | NS Pharma, Inc. | ||
NCT03167255 | Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) | Phase 2 Interventional | NS Pharma, Inc. |
External links ![]() |
For extended ADME data see the following: Drugs.com |