Eteplirsen
Orphan Drug Cold Chain RequiredFDA Approved
Contents
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 |
|---|---|---|---|---|
| — | — | — | — |
Buy / Source Eteplirsen — GNH India
7 matching products available through GNH India. Select a product to view details and request a quote on the GNH India website.
Exondys 51
50mg/ml x10mlvialsUSA
EXONDYS 51 100mg/2ml
100mg/2mlvialsIsrael
Exondys 100mg/2ml
100 mg/2 mLvialsIsrael
Exondys 10ml 51
500mg/10mLvialsUSA
Exondys 2ml 51
100 mg/2 mLvialsUSA
EXONDYS 100mg
100mg/2mlvialsUSA
EXONDYS 51 -500mg
500mg/10mlvialsUSA
Products and ordering are fulfilled by GNH India. You will be redirected to www.gnhindia.com.
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