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Viltolarsen: First Approval

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posted on 2020-06-09, 19:48 authored by figshare admin adisjournalsfigshare admin adisjournals
Compliance with Ethical Standards

Funding:The preparation of this review was not supported by any external funding.

Conflict of interest: During the peer review process the manufacturer of the agent under review was offered an opportunity to comment on the article. Changes resulting from any comments received were made by the authors on the basis of scientific completeness and accuracy. Anthony Markham is a contracted employee of Adis International Ltd/Springer Nature and is responsible for the article content and declare no relevant conflicts of interest.

Additional information about this Adis Drug Review can be found here


Viltolarsen (Viltepso® in Japan) is a phosphorodiamidate morpholino antisense oligonucleotide being developed by Nippon Shinyaku, in collaboration with the National Center of Neurology and Psychiatry (NCNP), for the treatment of Duchenne muscular dystrophy (DMD). Viltolarsen binds to exon 53 of the dystrophin mRNA precursor and restores the amino acid open-reading frame by skipping exon 53, resulting in the production of a shortened dystrophin protein that contains essential functional portions. In March 2020, intravenous viltolarsen received its first global approval in Japan for the treatment of DMD in patients with confirmed deletion of the dystrophin gene that is amenable to exon 53 skipping. Viltolarsen is under regulatory review in the USA and clinical trials continue in the USA, Canada and globally. This article summarizes the milestones in the development of viltolarsen leading to the first approval for DMD.

© Springer Nature Switzerland AG 2020


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