Viltolarsen: First Approval

  title={Viltolarsen: First Approval},
  author={Sohita Dhillon},
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… 
Pharmacological Profile of Viltolarsen for the Treatment of Duchenne Muscular Dystrophy: A Japanese Experience
The pharmacological profile of viltolarsen is summarized, focusing on the contribution of Japanese patients and researchers in its development, to produce truncated but partially functional dystrophin in DMD patients and restore muscle function.
CPAA_A_288842 235..242
Department of Medical Genetics, University of Alberta, Edmonton, Alberta, Canada Abstract: Duchenne muscular dystrophy (DMD) is a fatal, X-linked recessive disorder characterized by progressive
Intramuscular Evaluation of Chimeric Locked Nucleic Acid/2′OMethyl-Modified Antisense Oligonucleotides for Targeted Exon 23 Skipping in Mdx Mice
It is demonstrated that LNA/2′OMe AONs with a 30% LNA composition were significantly more potent in inducing exon skipping and dystrophin restoration in treated mdx muscles, compared to a previously tested 2′O-Methyl- and locked nucleic acid (LNA)-modified nucleotides with lower or higher LNA compositions.
Lessons Learned from Discontinued Clinical Developments in Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is an X-linked condition caused by a deficiency of functional dystrophin protein. Patients experience progressive muscle weakness, cardiomyopathy and have a
Structural Perturbations of Exon Skipping Edits within the Dystrophin D20:24 Region
A systematic and comprehensive panel of possible exon cuts in a region of the dystrophin protein is examined, and for the first time, exon edits that appear to maintain structural stability similar to wildtype protein are identified.
Developments in reading frame restoring therapy approaches for Duchenne muscular dystrophy
This work outlines developments for reading frame restoring approaches, challenges of mutation specificity, and optimizing delivery, and highlights ongoing efforts to better detect exon skipping therapeutic effects in clinical trials.
Utrophin modulator drugs as potential therapies for Duchenne and Becker muscular dystrophies
It is suggested that utrophin overexpression could act as a surrogate, compensating for the lack of dystrophin, and, as such, it could be used in combination with dyStrophin restoration therapies.
RNA Targeting in Inherited Neuromuscular Disorders: Novel Therapeutic Strategies to Counteract Mis-Splicing
Neuromuscular disorders represent multifaceted abnormal conditions, with little or no cure, leading to patient deaths from complete muscle wasting and atrophy. Despite strong efforts in the past
Therapeutic Strategies for Duchenne Muscular Dystrophy: An Update
The clinical potential of advanced strategies combining gene editing, cell-based therapy with tissue engineering, and myogenic cell transplantation for the treatment of muscular dystrophy are presented.
Muscle and cardiac therapeutic strategies for Duchenne muscular dystrophy: past, present, and future
A comprehensive and systematic review of literature on the gene, cell, and pharmacological experimental therapies aimed at restoring functional dystrophin or to counteract the associated processes contributing to disease progression like inflammation, fibrosis, calcium signaling or angiogenesis was carried out.


Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping
Results of this 4-week randomized clinical trial for safety followed by a 20-week open-label treatment period in 16 patients with DMD indicated significant drug-induced dystrophin production in both viltolarsen groups after 20 to 24 weeks of treatment.
Therapeutic developments for Duchenne muscular dystrophy
This Review outlines important developments in these research areas and specifically focuses on new therapies that are in the clinical trial phase or have already been approved for Duchenne muscular dystrophy.
An Overview of Recent Advances and Clinical Applications of Exon Skipping and Splice Modulation for Muscular Dystrophy and Various Genetic Diseases.
This chapter summarizes the development of antisense-mediated exon skipping therapy in diseases such as Usher syndrome, dystrophic epidermolysis bullosa, fibrodysplasia ossificans progressiva (FOP), and allergic diseases.
Systemic administration of the antisense oligonucleotide NS-065/NCNP-01 for skipping of exon 53 in patients with Duchenne muscular dystrophy
A phase 1 clinical trial of NS-065/NCNP-01 showed a favorable safety profile and pharmacokinetics, and the authors demonstrated that it effectively skipped exon 53 in the dystrophin gene, suggesting that a phase 2 trial of the drug is warranted.
Pharmacological advances for treatment in Duchenne muscular dystrophy.
Antisense Therapy in Neurology
This paper focuses on the current progress of antisense therapies in neurology and reports on the reported promising targets for antisense therapy.
Duchenne and Becker muscular dystrophy.
Duchenne and Becker muscular dystrophies have similar signs and symptoms and are caused by different mutations in the same gene. The two conditions differ in their severity, age of onset, and rate of
A Japanese phase I/II study of NS-065/NCNP-01, exon 53 skipping drug, in patients with Duchenne muscular dystrophy—a dose-finding study [abstract no. P.129 and poster
  • Neuromuscul Disord. 2018;28(Suppl 2):S68
  • 2018
Marketing authorization in Japan of VILTEPSO® intravenous infusion 250 mg for the treatment of Duchenne muscular dystrophy
  • [media release]
  • 2020