Eteplirsen: uses, dosing, side effects & brands
Eteplirsen is a medicine sold in the U.S. under one brand, for duchenne muscular dystrophy. Below: what the FDA label says, every product that contains it, what the pills look like, and its recall record.
Key facts
- Treats
- Duchenne Muscular Dystrophy
- Available as
- Injectable
- Sold as
- Exondys 51
- Prescription?
- Prescription only
- Generic available?
- Not in our catalog
How eteplirsen is dosed
From the FDA label for Exondys 51 (application NDA206488). Other eteplirsen products — different forms, different strengths — are dosed differently. Follow the label for the one you were prescribed.
30 milligrams per kilogram of body weight once weekly ( 2.1 ) Administer as an intravenous infusion over 35 to 60 minutes via an in-line 0.2 micron filter ( 2.1 , 2.3 ) Dilution required prior to administration ( 2.2 ) 2.1 Dosing Information The recommended dose of EXONDYS 51 is 30 milligrams per kilogram administered once weekly as a 35 to 60 minute intravenous infusion via an in-line 0.2 micron filter. If a dose of EXONDYS 51 is missed, it may be administered as soon as possible after the scheduled time. 2.2 Preparation Instructions EXONDYS 51 is supplied in single-dose vials as a preservative-free concentrated solution that requires dilution prior to administration. Parenteral drug products should be inspected visually for particulate matter and discoloration prior to administration, whenever solution and container permit. Use aseptic technique. Calculate the total dose of EXONDYS 51 to be administered based on the patient's weight and the recommended dose of 30 milligrams per kilogram. Determine the volume of EXONDYS 51 needed and the correct number of vials to supply the full calculated dose. Allow vials to warm to room temperature. Mix the contents of each vial by gently inverting 2 or 3 times. Do not shake. Visually inspect each vial of EXONDYS 51. EXONDYS 51 is a clear, colorless solution that may have some opalescence, and may contain white to off-white amorphous…
Eteplirsen side effects
The most common adverse reactions (incidence ≥35% and higher than placebo) were balance disorder and vomiting ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Sarepta Therapeutics, Inc. at 1-888-SAREPTA (1-888-727-3782) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. EXONDYS 51 was studied in a double-blind, placebo-controlled study for 24 weeks (Study 1), followed by an open label extension (Study 2). In Study 1, 12 patients were randomized to receive weekly intravenous infusions of EXONDYS 51 (n=8) or placebo (n=4) for 24 weeks. All 12 patients continued in Study 2 and received open-label EXONDYS 51 weekly for up to 208 weeks. In Study 1, 4 patients received placebo, 4 patients received EXONDYS 51 30 mg/kg, and 4 patients received EXONDYS 51 50 mg/kg (1.7 times the recommended dosage). In Study 2, 6 patients received EXONDYS 51 30 mg/kg/week and 6 patients received EXONDYS 51 50 mg/kg/week [see Clinical Studies ( 14 )] . Adverse reactions that occurred in 2 or more patients who received EXONDYS 51 and were more frequent than in the placebo group in Study 1 are presented in Table 1…
Who shouldn’t take eteplirsen
None. None ( 4 )
Every eteplirsen product we track (1)
Same active ingredient — different manufacturer, form, price and FDA recall record. That last one is what our independent score measures.
Only one: Exondys 51.
What people report to the FDA about eteplirsen
The FDA Adverse Event Reporting System (FAERS) collects reports from patients and clinicians. It holds 70 reports naming eteplirsen, and the FDA flagged 91% of those reports as serious. The effects reported most often — leaving out reports about overdose, misuse or the condition being treated, which dominate the raw list for common medicines:
- fall10 reports
- sepsis7 reports
- acute respiratory failure6 reports
- hypotension6 reports
- pneumonia6 reports
- respiratory failure5 reports
- covid-194 reports
- pneumonia aspiration4 reports
Read these as a signal, not a rate. A report does not mean eteplirsen caused the effect — anyone can file one, and many describe people taking several medicines for several conditions. Crucially there is no denominator: FAERS does not record how many people took the drug, so these counts cannot be turned into “X% of patients” — a bigger number often just means a more widely used or more talked-about drug. Duplicates exist, and publicity drives reporting. For what is actually established, read the FDA label section above.
Source: openFDA drug/event (FAERS), retrieved July 25, 2026.
Related calculators
Frequently asked questions
What is eteplirsen?
EXONDYS 51 (eteplirsen) injection is a sterile, aqueous, preservative-free, concentrated solution for dilution prior to intravenous administration. EXONDYS 51 is clear and colorless, and may have some opalescence, and may contain white to off-white amorphous particles. EXONDYS 51 is supplied in single dose vials containing 100 mg or 500 mg eteplirsen (50 mg/mL). EXONDYS 51 is formulated as an isotonic, phosphate buffered saline solution with an osmolality of 260 to 320 mOsm and a pH of 7.5. Each milliliter of EXONDYS 51 contains 50 mg eteplirsen; 0.2 mg potassium chloride, 0.2 mg potassium phosphate monobasic, 8 mg sodium chloride, and 1.14 mg sodium phosphate dibasic, anhydrous, in water for injection. The product may contain hydrochloric acid or sodium hydroxide to adjust pH. Eteplirsen is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer (PMO) subclass. PMOs are synthetic molecules in which the five-membered ribofuranosyl rings found in natural DNA and RNA are replaced by a six-membered morpholino ring. Each morpholino ring is linked through an uncharged phosphorodiamidate moiety rather than the negatively charged phosphate linkage that is present in natural DNA and RNA. Each phosphorodiamidate morpholino subunit contains one of the heterocyclic bases found in DNA (adenine, cytosine, guanine, or thymine). Eteplirsen contains 30 linked subunits. The…
Can you take eteplirsen with other medicines?
It depends on the medicine. We check it against the FDA labels rather than guessing: our interaction checker searches each drug's own label for the other and quotes what it says, naming the section it came from. Run eteplirsen against whatever else you take — and remember that a label not naming a drug is not the same as that combination being safe.
What forms does eteplirsen come in?
Across the brands we track, eteplirsen is currently marketed as injectable, per the FDA's National Drug Code Directory. Each form is dosed differently — follow the label for the exact product you were prescribed.
Is there a generic eteplirsen?
We do not currently list a generic-labelled eteplirsen product. That does not always mean none exists — it means none appears under a generic name in the FDA data we track. Ask your pharmacist.
Cite this page
- APA
- pharmaranks. (2026, July 24). Eteplirsen: uses, dosing, side effects & brands. https://pharmaranks.com/drugs/eteplirsen
- MLA
- “Eteplirsen: uses, dosing, side effects & brands.” pharmaranks, 24 July 2026, https://pharmaranks.com/drugs/eteplirsen.
We summarise public FDA and NIH sources — for a clinical claim, cite the primary source we link to as well.
Sources: FDA openFDA drug label, National Drug Code Directory, and Enforcement (recall) database. This page reproduces public FDA data and is not medical advice. Dosing is set by your prescriber.
Read the full FDA label for eteplirsen on DailyMed (NIH) ↗.