Eteplirsen
Orphan Drug Cold Chain RequiredFDA Approved
Description
Eteplirsen is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation amenable to exon 51 skipping. It promotes exon skipping during mRNA processing, leading to the production of a truncated but functional dystrophin protein.
Indications & Therapeutic Use
Duchenne muscular dystrophy (DMD) with confirmed mutation amenable to exon 51 skipping
Linked Diseases:
Global Availability (1 countries)
| Country | Access Route | Regulatory Pathway | Lead Time | Status |
|---|---|---|---|---|
| — | — | — | — |
Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
Eteplirsen
| Generic Name | Eteplirsen |
| Brands | 1 brand available |
| Active Ingredient | Eteplirsen |
| Drug Class | Duchenne muscular dystrophy (DMD) with confirmed mutation amenable to exon 51 skipping |
| Manufacturer | Sarepta Therapeutics |
| Dosage Forms | IV infusion |
| Medical Code | M09AX09 |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Required |
| Reg. Status | FDA Approved |
| Clinical Trial | NCT01599091 |
| Countries | 1 countries |
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations2 Validated Nodes