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Molecule

Eteplirsen

Antisense Oligonucleotide
Available on requestOrphanCold chainRare diseases and enzyme replacement
Presentations
2
Brands
1
Manufacturers
1
Available from stock
0

Eteplirsen is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. MedsAcross supplies Eteplirsen for export from India to licensed hospitals, importers and physicians acting for named patients, with the documents each country requires.

Information from the US FDA prescribing information, retrieved 12 November 2026. Not yet medically reviewed.How we review

Key facts

Pharmacological class
Antisense Oligonucleotide, Oligonucleotides
ATC classification
M09AX06 Eteplirsen · Other musculoskeletal medicines, subgroup M09A, subgroup M09AX
US FDA first approval
19 September 2016
EU status
Refused
Orphan designation
Yes
WHO essential medicine
Not recorded
India (CDSCO)
Not yet confirmed against CDSCO approval lists
Routes
Intravenous
Dosage forms
Injection
Identifiers
UNII AIW6036FASRxCUI 1810569CAS 1173755-55-9DrugBank DB06014Q5402585

Clinical information

From the regulator-approved product information. Each section names its source.

Uses

EXONDYS 51 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with EXONDYS 51. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. EXONDYS 51 is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping.

Source: US FDA prescribing information

How it works

Eteplirsen is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein, which was evaluated in Study 2 and Study 3.

Source: US FDA prescribing information

Dosing overview

30 milligrams per kilogram of body weight once weekly. Administer as an intravenous infusion over 35 to 60 minutes via an in-line 0.2 micron filter. Dilution required prior to administration

Source: US FDA prescribing information

Warnings and precautions

Hypersensitivity Reactions: Hypersensitivity reactions, including bronchospasm, chest pain, cough, tachycardia, and urticaria, have occurred in patients treated with EXONDYS 51. If hypersensitivity reactions occur, institute appropriate medical treatment and consider slowing the infusion or interrupting the EXONDYS 51 therapy.

Source: US FDA prescribing information

Side effects

The most common adverse reactions (incidence ≥35% and higher than placebo) were balance disorder and vomiting

Source: US FDA prescribing information

Storage

Store EXONDYS 51 at 2°C to 8°C (36°F to 46°F). Do not freeze. Protect from light and store EXONDYS 51 in the original carton until ready for use.

Source: US FDA prescribing information

For healthcare professionals and buyers. This is not medical advice. Treatment decisions belong to the prescriber.

All presentations

2 presentations of Eteplirsen, sorted by availability.

MedicineManufacturerRegisterAvailability
Exondys 51 100 mg/2 mL injectionIntravenous Cold chainSarepta TherapeuticsUS FDAAvailable on request
Exondys 51 500 mg/10 mL injectionIntravenous Cold chainSarepta TherapeuticsUS FDAAvailable on request

Brands by market

1 brand names across 1 markets.

Brand names by country

United States

Exondys 51

Manufacturers

Regulatory status

Approval facts from US FDA, EMA and CDSCO data, and registration status by country where we hold register data.

RegisterStatusSource
US FDAFirst approved 19 September 2016Drugs@FDA
EU (EMA)RefusedEMA medicines data
India (CDSCO)Not yet confirmed against CDSCO approval lists—
United StatesRegisteredUS FDA (NDC directory, openFDA)Checked 12 November 2026

Conditions

Conditions linked to Eteplirsen in public data. This is not a list of approved uses, see the clinical information.

Duchenne muscular dystrophyRare disease

Questions and answers

Is Eteplirsen available for export from India?

Yes, on request. MedsAcross lists 2 presentations of Eteplirsen and sources them for licensed hospitals, importers and physicians against valid import documents.

What brand names does Eteplirsen have?

The catalogue lists 1 brand names for Eteplirsen, including Exondys 51.

Is Eteplirsen approved by the US FDA?

Yes. The US FDA first approved Eteplirsen on 19 September 2016, according to Drugs@FDA.

Does Eteplirsen need cold chain shipping?

At least one listed presentation of Eteplirsen must be kept cold, so it is shipped in temperature-controlled packaging. Check the storage line on each presentation.

Which ATC class is Eteplirsen in?

Eteplirsen is classified under ATC code M09AX06 (Eteplirsen).

References

  1. Exondys 51 prescribing information (Sarepta Therapeutics). U.S. National Library of Medicine, DailyMed. Retrieved 13 August 2025

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