Viltolarsen
| Clinical data | |
|---|---|
| Trade names | Viltepso |
| Other names | NS-065/NCNP-01 |
| AHFS/Drugs.com | Monograph |
| License data |
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| Routes of administration | Intravenous |
| Drug class | Antisense oligonucleotide |
| ATC code | |
| Legal status | |
| Legal status |
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| Identifiers | |
| CAS Number | |
| DrugBank | |
| ChemSpider | |
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| Chemical and physical data | |
| Formula | C244H381N113O88P20 |
| Molar mass | 6924.910 g·mol−1 |
Viltolarsen, sold under the brand name Viltepso, is a medication used for the treatment of Duchenne muscular dystrophy (DMD). Viltolarsen is a Morpholino antisense oligonucleotide.
The most common side effects include upper respiratory tract infection, injection site reaction, cough, and pyrexia (fever).
Viltolarsen was approved for medical use in the United States in August 2020. After golodirsen was approved in December 2019, viltolarsen is the second approved targeted treatment for people with this type of mutation in the United States. Approximately 8% of people with DMD have a mutation that is amenable to exon 53 skipping.