The FDA has granted accelerated approval for Viltepso (viltolarsen) injection for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the DMD gene is amenable to exon 53 skipping.
The early clinical data demonstrated an increase in dystrophin production that is reasonably likely to predict clinical benefit. NS Pharma is now conducting their Phase III trial to confirm a clinical benefit. This could be the second exon-skipping therapy for individuals with mutations that are amenable to exon 53. Sarepta’s golodirsen is already approved in the USA. Sarepta also has eteplirsen approved for exon 51 amenable mutations and are working on casimersen for exon 45 amenable mutations.
This is excellent news for our families in the United States. However, it is a heavy reminder for Canadians that we still wait for any of these treatments to come to Canada.
Clinical trials or Health Canada’s Special Access Program remain the only way Canadians can gain early access to any of these medications.
There is currently one trial site open in Canada for vitolarsen at Alberta Children’s Hospital. For more information, speak to your neuromuscular team.
If you are interested in learning more about NS Pharma’s early access program, please contact NS Pharma at 1-877-539-8515, DMDResearch@NSPharma.com.
If you have any questions regarding this information, contacting the company, or clinical trials in general, please reach out to:
Director of Advocacy and Research
Defeat Duchenne Canada