Viltolarsen is a Antisense Oligonucleotide owned by Nippon Shinyaku, and is involved in 9 clinical trials, of which 5 were completed, and 4 are ongoing. Viltolarsen (NS-065) is based on the exon skipping technology which skips exon 53. Exon skipping is a therapeutic approach based on the use of a synthetic oligonucleotide, called an antisense oligonucleotide, to restore the amino acid reading frame by artificially skipping certain exons of the transcription product (mRNA) to be translated into protein. Then Exon 51 can now join up to exon 54 and continue to make the rest of the protein, with exons 52 and 53 missing in the middle. Therefore, exon 53 skipping by NS-065 (NCNP-01) be able to reduce the symptoms of Duchenne muscular dystrophy.

The revenue for Viltolarsen is expected to reach a total of $3.7bn through 2038. This change impacts the valuation of this asset and is an important factor to understand the current value of the drug in a clinical process. View the complete picture with the Viltolarsen NPV Report.

Viltolarsen was originated by National Center of Neurology and Psychiatry and Nippon Shinyaku and is currently owned by Nippon Shinyaku.

Viltolarsen Overview

Viltepso is formulated as solution for intravenous route of administration. Viltepso is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. Viltolarsen (NS-065) is under development for the treatment of Duchenne muscular dystrophy (DMD). It acts by targeting Dystrophin.

Nippon Shinyaku Overview

Nippon Shinyaku develops, manufactures, and sells ethical pharmaceuticals and functional foods. The company offers a wide range of products including drugs for pain, inflammation, and allergies; urological diseases; hematologic malignancies; gastrointestinal disorders; cardiovascular and metabolic diseases among others. Nippon Shinyaku also provides functional food ingredients including health food ingredients, preservatives, spices and condiments, and protein preparations. Its functional food products find application in meat processing, fish processing, dairy product, prepared food, confectionery and bakery, and beverage, among others. The company operates business through a network of offices and research laboratories located in Japan, China, the UK and the US. Nippon Shinyaku is headquartered in Minami-ku, Kyoto, Japan.

The company reported revenues of (Yen) JPY137,484 million for the fiscal year ended March 2022 (FY2022), an increase of 12.8% over FY2021. In FY2022, the company’s operating margin was 20.6%, compared to an operating margin of 21.4% in FY2021. In FY2022, the company recorded a net margin of 16.8%, compared to a net margin of 17% in FY2021. The company reported revenues of JPY35,517 million for the second quarter ended September 2022, a decrease of 0.3% over the previous quarter.

Quick View – Viltolarsen

Report Segments
  • Innovator (NME)
Drug Name
  • Viltolarsen
Administration Pathway
  • Intravenous
Therapeutic Areas
  • Genetic Disorders
Key Companies
Highest Development Stage
  • Marketed

GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

To create this model, GlobalData takes into account factors including patent law, known and projected regulatory approval processes, cash flows, potential applicable patients, drug margins, company expenses, and pricing estimates. Combining these data points with GlobalData’s world class analysis creates high value models that companies can use to help in evaluation processes for each drug or company.

The rNPV method integrates the probability of a drug reaching a clinical stage into the cash flow at that time, which provides a more accurate rNPV, as it considers the probability that the drug never makes it through the clinical pathway to commercialization. GlobalData’s rNPV model uses proprietary likelihood of approval (LoA)and phase transition success rate(PTSR) data for the indication in the highest development stage, which can be found on GlobalData’s Pharmaceutical Intelligence Center.