The European Commission has granted orphan drug designation to viltolarsen, a treatment for people with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping.

See the June 8, 2020, vilolarsen press release here.

This compound was approved in Japan in March this year under the brand name Viltepso and is currently under priority review by the FDA in the USA. It is not known what the plans are for Canada, which still has no specific orphan drug framework. This compound is being developed by Nippon Shinyaku and its subsidiary in the US NS pharma.

The phase three clinical trial is currently recruiting ambulatory boys 4-7 years old. There is presently one Canadian site listed in Calgary.

For more information on locations and eligibility, we encourage you to speak to your neuromuscular team and go to:

• www.clinicaltrials.gov
• Search for NCT04060199

US NS Pharma: www.nspharma.com

Nippon Shinyaku: www.nippon-shinyaku.co.jp/english/