There are currently four exon skipping medications approved by the FDA in the USA.
Unfortunately, none of these are approved in Canada and all exon therapies are considered investigational by Health Canada. Understandably, this has been very frustrating for our community, but Defeat Duchenne Canada is advocating for change. Learn more about our advocacy work.
The table below summarizes all exon therapies and where they are in the drug development process. Try our Clinical Trial Finder Tool to see if a trial is right for you.
Drug Name | Type of Drug | DMD Mutations Addressed | Sponsor | Current Development Phase | Approval Status |
|---|---|---|---|---|---|
Casimersen | IV infusion 30mg/kg once weekly | Exon 45 skipping includes deletions of: 7-44, 12-44, 18-44, 44, 46, 46-47, 46-48, 46-49, 46-51, 46-53, 46-55, 46-57, 46-59, 46-60, 46-67, 46-69, 46-75, 46-78 | Sarepta | Phase 3 programs are still ongoing | AMONDYS 45 in USA |
Eteplirsen | IV infusion 30mg/kg once weekly | Exon 51 skipping includes deletions of: 3-50, 4-50, 5-50, 6-50, 9-50, 10-50, 11-50, 13-50, 14-50, 15-50, 16-50, 17-50, 19-50, 21-50, 23-50, 24-50, 25-50, 26-50, 27-50, 28-50, 29-50, 30-50, 31-50, 32-50, 33-50, 34-50, 35-50, 36-50, 37-50, 38-50, 39-50, 40-50, 41-50, 42-50, 43-50, 45-50, 47-50, 48-50, 49-50, 50, 52, 52-58, 52-61, 52-63, 52-64, 52-66, 52-76, 52-77 | Sarepta | Phase 3 programs are still ongoing | EXONDYS 51 in USA |
Golodirsen | IV infusion 30mg/kg once weekly | Exon 53 skipping includes deletions of: 3-52, 4-52, 5-52, 6-52, 9-52, 10-52, 11-52, 13-52, 14-52, 15-52, 16-52, 17-52, 19-52, 21-52, 23-52, 24-52, 25-52, 26-52, 27-52, 28-52, 29-52, 30-52, 31-52, 32-52, 33-52, 34-52, 35-52, 36-52, 37-52, 38-52, 39-52, 40-52, 41-52, 42-52, 43-52, 45-52, 47-52, 48-52, 49-52, 50-52, 52, 54-58, 54-61, 54-63, 54-64, 54-66, 54-76, 54-77 | Sarepta | Phase 3 programs are still ongoing | VYONDYS 53 in USA |
SRP-5051 | IV infusion given once monthly | Exon 51 skipping includes deletions of: 3-50, 4-50, 5-50, 6-50, 9-50, 10-50, 11-50, 13-50, 14-50, 15-50, 16-50, 17-50, 19-50, 21-50, 23-50, 24-50, 25-50, 26-50, 27-50, 28-50, 29-50, 30-50, 31-50, 32-50, 33-50, 34-50, 35-50, 36-50, 37-50, 38-50, 39-50, 40-50, 41-50, 42-50, 43-50, 45-50, 47-50, 48-50, 49-50, 50, 52, 52-58, 52-61, 52-63, 52-64, 52-66, 52-76, 52-77 | Sarepta | Phase 2 | Still under investigation |
Viltolarsen | IV infusion 80 mg/kg once weekly | Exon 53 skipping includes deletions of: 3-52, 4-52, 5-52, 6-52, 9-52, 10-52, 11-52, 13-52, 14-52, 15-52, 16-52, 17-52, 19-52, 21-52, 23-52, 24-52, 25-52, 26-52, 27-52, 28-52, 29-52, 30-52, 31-52, 32-52, 33-52, 34-52, 35-52, 36-52, 37-52, 38-52, 39-52, 40-52, 41-52, 42-52, 43-52, 45-52, 47-52, 48-52, 49-52, 50-52, 52, 54-58, 54-61, 54-63, 54-64, 54-66, 54-76, 54-77 | NS Pharma | Phase 2, 3 and 4 studies still ongoing | VILTEPSO 53 in USA |
DS-5141b | Subcutaneous injection once weekly | Exon 45 skipping includes deletions of: 7-44, 12-44, 18-44, 44, 46, 46-47, 46-48, 46-49, 46-51, 46-53, 46-55, 46-57, 46-59, 46-60, 46-67, 46-69, 46-75, 46-78 | Daiichi Sankyo | Phase 2 | Still under investigation |
AOC 1044 | Unknown at this time | Exon 44 skipping includes deletions of: 3-43, 4-43, 5-43, 6-43, 9-43, 10-43, 11-43, 13-43, 14-43, 15-43, 16-43, 17-43, 19-43, 21-43, 23-43, 24-43, 25-43, 26-43, 27-43, 28-43, 29-43, 30-43, 31-43, 32-43, 33-43, 34-43, 35-43, 36-43, 37-43, 38-43, 39-43, 40-43, 41-43, 42-43, 43, 45, 45-54, 45-56, 45-62, 45-65, 45-68, 45-70, 45-71, 45-72, 45-73, 45-74 | Avidity Biosciences | Clinical trial initiations planned for 2022 | Still under investigation |
DYNE-251 | Unknown at this time | Exon 51 skipping includes deletions of: 3-50, 4-50, 5-50, 6-50, 9-50, 10-50, 11-50, 13-50, 14-50, 15-50, 16-50, 17-50, 19-50, 21-50, 23-50, 24-50, 25-50, 26-50, 27-50, 28-50, 29-50, 30-50, 31-50, 32-50, 33-50, 34-50, 35-50, 36-50, 37-50, 38-50, 39-50, 40-50, 41-50, 42-50, 43-50, 45-50, 47-50, 48-50, 49-50, 50, 52, 52-58, 52-61, 52-63, 52-64, 52-66, 52-76, 52-77 | Dyne Therapeutics | Clinical trial initiations planned for 2022 | Still under investigation |
PGN-ED051 | Unknown at this time | Exon 51 skipping includes deletions of: 3-50, 4-50, 5-50, 6-50, 9-50, 10-50, 11-50, 13-50, 14-50, 15-50, 16-50, 17-50, 19-50, 21-50, 23-50, 24-50, 25-50, 26-50, 27-50, 28-50, 29-50, 30-50, 31-50, 32-50, 33-50, 34-50, 35-50, 36-50, 37-50, 38-50, 39-50, 40-50, 41-50, 42-50, 43-50, 45-50, 47-50, 48-50, 49-50, 50, 52, 52-58, 52-61, 52-63, 52-64, 52-66, 52-76, 52-77 | PepGen | Clinical trial initiations planned for 2022 | Still under investigation |