DMD: a drug targeting exon-skipping of exon 53 authorized in Japan

The Japanese laboratory Shinyaku Co. Ltd announced on March 25, 2020 by press release the commercial approval in Japan of its antisense oligonucleotide targeting exon-skipping of exon 53 of the dystrophin gene (DMD gene), viltolarsen (NS- 065 / NCNP-01- Viltepso), in Duchenne muscular dystrophy. Viltolarsen 250 mg is given by intravenous injection, once a week, at a dose of 80 mg / kg.

The viltolarsen developed by the laboratory Shinyaku Co. Ltd and the National Center of neurology and Psychiatry, comes to enlarge the family of antisense oligonucleotides having already received a marketing authorization in DMD, Exondys51 and Vyondys53, this time to United States. Viltolarsen may also be prescribed in the United States as part of an expanded acces program.

Japanese health authorities have taken into account the results of two completed viltolarsen clinical trials: a phase I trial in 10 boys with DMD aged 5 to 18 years and a double-blind phase I / II trial against placebo in 16 boys with DMD aged 4 to 9 years old for 1.5 years.

The laboratory did not provide details on these results. An international phase III trial (RACER53) has just started to confirm these data in the longer term and with a larger cohort.


Nippon Shinyaku Co. Ltd press release, March 25, 2020 “Marketing authorization in Japan of VILTEPSO® Intravenous Infusion 250 mg for the treatment of Duchenne muscular dystrophy”