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Exondys 51 — Competitive Intelligence Brief
marketed
Antisense Oligonucleotide [EPC]
exon 51 of dystrophin pre-mRNA
Rare Disease
Oligonucleotide
Live · refreshed every 30 min
Target snapshot
Exondys 51 (ETEPLIRSEN) — Sarepta. Exondys 51 binds to specific RNA sequences to prevent the skipping of exon 51 during dystrophin mRNA splicing.
Comparator set (6 drugs)
| Drug | Generic | Sponsor | Phase | Class | Target | Approval / PDUFA |
|---|---|---|---|---|---|---|
| Exondys 51 TARGET | ETEPLIRSEN | Sarepta | marketed | Antisense Oligonucleotide [EPC] | exon 51 of dystrophin pre-mRNA | 2016-01-01 |
| Qalsody | TOFERSEN | Biogen Ma | marketed | Antisense Oligonucleotide [EPC] | Superoxide dismutase 1 (SOD1) mRNA | 2023-01-01 |
| Amondys 45 | CASIMERSEN | Sarepta | marketed | Antisense Oligonucleotide [EPC] | exon 53 of dystrophin pre-mRNA | 2021-01-01 |
| Vyondys 53 | GOLODIRSEN | Sarepta | marketed | Antisense Oligonucleotide [EPC] | exon 53 of dystrophin pre-mRNA | 2019-01-01 |
| Kynamro | MIPOMERSEN | Kastle Theraps Llc | marketed | Antisense Oligonucleotide | 2013-01-01 | |
| PF-07852352 Influenza saRNA 1 | pf-07852352-influenza-sarna-1 | Pfizer | marketed | antisense oligonucleotide | influenza virus | |
| ISIS 3521 | ISIS 3521 | European Organisation for Research and Treatment of Cancer - EORTC | phase 3 | Antisense oligonucleotide | Protein kinase C-alpha (PKC-alpha) mRNA |
Recent regulatory actions (last 90 days)
Upcoming PDUFA dates (next 180 days)
Patent timeline (forward window)
Sponsor landscape (Antisense Oligonucleotide [EPC] class)
- Sarepta · 3 drugs in this class
- Biogen Ma · 1 drug in this class
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Cite this brief
Drug Landscape (2026). Exondys 51 — Competitive Intelligence Brief. https://druglandscape.com/ci/eteplirsen. Accessed 2026-05-14.
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