Eteplirsen side effects: what 2,391 FDA adverse event reports show

Eteplirsen · Exondys 51 · 27 reaction terms · 2,350 drug–reaction co-reports · data 2026-09-14

Drug class

Antisense Oligonucleotide

FDA label (Established Pharmacologic Class) · compare the class

Brand names

Exondys 51

openFDA label

Who reports

2,391 reports · 20% serious · 66 with death outcome · 2% female

openFDA FAERS counts, all years, deduplication not applied

Used for

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 .

FDA label, Indications and Usage

What it is

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.

FDA label, Description section

Boxed warning

None in the current label

How to read this page. FAERS is a spontaneous-reporting system: counts are not incidence rates and a signal is not proof of causation. Each reaction below is run through an automated screen — is the term in the FDA label (known), in the indications (the disease being treated), an administrative/coding term, or none of these (unlabeled)? That screen is mechanical text matching, not a causality assessment. The 30 hand-written Bradford Hill assessments are on the causality assessment page. Not medical advice.

Causality screen of the 19 strongest signals

Reporting / coding artifact 9Not in label — unassessed 10

What the badges mean

Reporting / coding artifact An administrative, monitoring or exposure term (product issue, off-label use, 'test normal', overdose...). Not a biological reaction; a high PRR here reflects how reports are coded.
Not in label — unassessed Not found in the label text, not an artifact pattern. This is a genuine candidate for a formal causality assessment (Bradford Hill / WHO-UMC); it has NOT had one here.

Strongest disproportionality signals

Flagged terms (PRR ≥ 2, χ² ≥ 4, n ≥ 10), ranked by PRR; infinite PRRs (no comparator reports) excluded. The EBGM column is a second, shrinkage-based estimate (MGPS) with its 5th percentile EB05 and the BCPNN IC025; a pair flagged by PRR alone is marked PRR-only and should be read as fragile. The strata column recomputes the PRR on serious reports only and flags pairs whose reports are unusually skewed by sex or come mostly from consumers rather than clinicians. 8 further terms are in the interactive explorer. Reaction links open the term's cross-drug page.

Show: hide coding/lab/outcome terms hide indication terms unlabeled only hide PRR-only

Reaction	Reports	PRR 95% CI · ROR · χ²/p	EBGM EB05 · IC025	Label screen	Serious-only & strata	Trial cross-check
Poor venous access	187	161.8 140.8–185.9ROR 175.5 · χ² 29,195 / p <1e-300	156.4 EB05 138.4 · IC025 +6.6 3/3 methods agree	Not in label — unassessed	serious n=4	no trial data cached
Intentional dose omission	279	90.4 80.9–101.0ROR 102.2 · χ² 24,353 / p <1e-300	88.9 EB05 80.5 · IC025 +6.1 3/3 methods agree	Reporting / coding artifact	serious PRR 9.5 · 96% clinician	no trial data cached
No adverse event	550	27.3 25.3–29.4ROR 35.1 · χ² 13,971 / p <1e-300	27.2 EB05 25.3 · IC025 +4.6 3/3 methods agree	Reporting / coding artifact	97% clinician	no trial data cached
Product dose omission issue	696	21.5 20.2–22.9ROR 29.9 · χ² 13,729 / p <1e-300	21.4 EB05 20.1 · IC025 +4.3 3/3 methods agree	Reporting / coding artifact	serious PRR 12.8	no trial data cached
Device issue	182	18.3 15.9–21.1ROR 19.8 · χ² 2,971 / p <1e-300	18.3 EB05 16.1 · IC025 +3.9 3/3 methods agree	Reporting / coding artifact	serious PRR 5.5 · 90% clinician	no trial data cached
Gastroenteritis viral	18	8.7 5.5–13.8ROR 8.8 · χ² 115 / p 3e-27	7.6 EB05 4.9 · IC025 +2.1 3/3 methods agree	Not in label — unassessed	serious PRR 10.6	no trial data cached
Insurance issue	14	8.3 4.9–13.9ROR 8.3 · χ² 82 / p 6e-20	6.9 EB05 4.2 · IC025 +1.8 3/3 methods agree	Not in label — unassessed	–	no trial data cached
Device occlusion	13	8.2 4.8–14.1ROR 8.2 · χ² 75 / p 2e-18	6.7 EB05 4.1 · IC025 +1.8 3/3 methods agree	Reporting / coding artifact	serious n=1	no trial data cached
Pharyngitis streptococcal	10	8.1 4.4–15.1ROR 8.1 · χ² 55 / p 5e-14	6.3 EB05 3.5 · IC025 +1.5 3/3 methods agree	Not in label — unassessed	serious n=1	no trial data cached
Acute respiratory failure	15	6.9 4.2–11.4ROR 6.9 · χ² 70 / p 4e-17	5.8 EB05 3.7 · IC025 +1.7 3/3 methods agree	Not in label — unassessed	serious PRR 19.9	no trial data cached
Femur fracture	24	6.8 4.6–10.1ROR 6.8 · χ² 113 / p 1e-26	6.1 EB05 4.3 · IC025 +1.9 3/3 methods agree	Not in label — unassessed	serious PRR 18.9	no trial data cached
Product dose omission	41	5.4 4.0–7.4ROR 5.5 · χ² 144 / p 2e-33	5.1 EB05 3.9 · IC025 +1.8 3/3 methods agree	Reporting / coding artifact	serious PRR 14.1 · 93% clinician	no trial data cached
Illness	55	4.8 3.7–6.2ROR 4.9 · χ² 163 / p 2e-37	4.6 EB05 3.7 · IC025 +1.8 3/3 methods agree	Reporting / coding artifact	serious PRR 1.6	no trial data cached
Increased appetite	10	4.7 2.5–8.7ROR 4.7 · χ² 25 / p 2e-7	3.9 EB05 2.3 · IC025 +0.9 3/3 methods agree	Not in label — unassessed	serious PRR 10.5	no trial data cached
Lower limb fracture	10	4.6 2.5–8.5ROR 4.6 · χ² 25 / p 3e-7	3.9 EB05 2.3 · IC025 +0.9 3/3 methods agree	Not in label — unassessed	serious PRR 11.7	no trial data cached
Covid-19	87	4.3 3.5–5.3ROR 4.4 · χ² 220 / p 5e-50	4.2 EB05 3.5 · IC025 +1.7 3/3 methods agree	Not in label — unassessed	serious PRR 4.6	no trial data cached
Influenza	52	4.1 3.1–5.3ROR 4.1 · χ² 118 / p 7e-28	3.9 EB05 3.1 · IC025 +1.5 3/3 methods agree	Not in label — unassessed	serious PRR 5.3	no trial data cached
Underdose	33	4.0 2.8–5.6ROR 4.0 · χ² 70 / p 2e-17	3.8 EB05 2.8 · IC025 +1.3 3/3 methods agree	Reporting / coding artifact	97% clinician	no trial data cached
Therapy cessation	23	3.9 2.6–5.9ROR 4.0 · χ² 48 / p 2e-12	3.7 EB05 2.6 · IC025 +1.2 3/3 methods agree	Reporting / coding artifact	serious PRR 3.3 · 91% clinician	no trial data cached

Trial evidence (ClinicalTrials.gov)

Trial cross-check not yet run for this drug (ClinicalTrials.gov pull covers the 800 most-reported drugs first).

Most frequently reported reactions

By raw co-report count, regardless of disproportionality. Common terms here are often the illness itself or generic outcomes.

Reaction	Reports	PRR 95% CI · ROR · χ²/p	EBGM EB05 · IC025	Label screen	Serious-only & strata	Trial cross-check
Product dose omission issue	696	21.5 20.2–22.9ROR 29.9 · χ² 13,729 / p <1e-300	21.4 EB05 20.1 · IC025 +4.3 3/3 methods agree	Reporting / coding artifact	serious PRR 12.8	no trial data cached
No adverse event	550	27.3 25.3–29.4ROR 35.1 · χ² 13,971 / p <1e-300	27.2 EB05 25.3 · IC025 +4.6 3/3 methods agree	Reporting / coding artifact	97% clinician	no trial data cached
Intentional dose omission	279	90.4 80.9–101.0ROR 102.2 · χ² 24,353 / p <1e-300	88.9 EB05 80.5 · IC025 +6.1 3/3 methods agree	Reporting / coding artifact	serious PRR 9.5 · 96% clinician	no trial data cached
Poor venous access	187	161.8 140.8–185.9ROR 175.5 · χ² 29,195 / p <1e-300	156.4 EB05 138.4 · IC025 +6.6 3/3 methods agree	Not in label — unassessed	serious n=4	no trial data cached
Device issue	182	18.3 15.9–21.1ROR 19.8 · χ² 2,971 / p <1e-300	18.3 EB05 16.1 · IC025 +3.9 3/3 methods agree	Reporting / coding artifact	serious PRR 5.5 · 90% clinician	no trial data cached
Covid-19	87	4.3 3.5–5.3ROR 4.4 · χ² 220 / p 5e-50	4.2 EB05 3.5 · IC025 +1.7 3/3 methods agree	Not in label — unassessed	serious PRR 4.6	no trial data cached
Illness	55	4.8 3.7–6.2ROR 4.9 · χ² 163 / p 2e-37	4.6 EB05 3.7 · IC025 +1.8 3/3 methods agree	Reporting / coding artifact	serious PRR 1.6	no trial data cached
Influenza	52	4.1 3.1–5.3ROR 4.1 · χ² 118 / p 7e-28	3.9 EB05 3.1 · IC025 +1.5 3/3 methods agree	Not in label — unassessed	serious PRR 5.3	no trial data cached
Product dose omission	41	5.4 4.0–7.4ROR 5.5 · χ² 144 / p 2e-33	5.1 EB05 3.9 · IC025 +1.8 3/3 methods agree	Reporting / coding artifact	serious PRR 14.1 · 93% clinician	no trial data cached
Underdose	33	4.0 2.8–5.6ROR 4.0 · χ² 70 / p 2e-17	3.8 EB05 2.8 · IC025 +1.3 3/3 methods agree	Reporting / coding artifact	97% clinician	no trial data cached
Femur fracture	24	6.8 4.6–10.1ROR 6.8 · χ² 113 / p 1e-26	6.1 EB05 4.3 · IC025 +1.9 3/3 methods agree	Not in label — unassessed	serious PRR 18.9	no trial data cached
Therapy cessation	23	3.9 2.6–5.9ROR 4.0 · χ² 48 / p 2e-12	3.7 EB05 2.6 · IC025 +1.2 3/3 methods agree	Reporting / coding artifact	serious PRR 3.3 · 91% clinician	no trial data cached
Gastroenteritis viral	18	8.7 5.5–13.8ROR 8.8 · χ² 115 / p 3e-27	7.6 EB05 4.9 · IC025 +2.1 3/3 methods agree	Not in label — unassessed	serious PRR 10.6	no trial data cached
Acute respiratory failure	15	6.9 4.2–11.4ROR 6.9 · χ² 70 / p 4e-17	5.8 EB05 3.7 · IC025 +1.7 3/3 methods agree	Not in label — unassessed	serious PRR 19.9	no trial data cached
Insurance issue	14	8.3 4.9–13.9ROR 8.3 · χ² 82 / p 6e-20	6.9 EB05 4.2 · IC025 +1.8 3/3 methods agree	Not in label — unassessed	–	no trial data cached

Unlabeled candidate signals

These flagged terms were not found in the FDA label text and do not match an artifact pattern. They are the pairs that would merit a structured assessment (temporality, dose-response, biological plausibility, consistency across databases, trial or cohort evidence). The screen has not done that; string-matching misses label wording, and a strong PRR can still be stimulated reporting, co-medication, or the disease itself.

Poor venous access — n=187, PRR 161.8
Femur fracture — n=24, PRR 6.8
Covid-19 — n=87, PRR 4.3
Influenza — n=52, PRR 4.1

Want to assess one of these properly? The runbook describes the Bradford Hill / trial-evidence workflow used on the assessment page, and handoff_top1000_signals.csv lists the strongest unassessed pairs across all drugs.

Common questions

What are the most reported side effects of Eteplirsen?

In FDA FAERS, the most frequently co-reported reaction terms for Eteplirsen are poor venous access, covid-19, influenza, femur fracture, gastroenteritis viral, acute respiratory failure. These are report counts, not rates: FAERS has no denominator, reporting is voluntary and uneven, and a term can be reported because of the illness being treated rather than the drug.

Does Eteplirsen have an FDA boxed warning?

No. The FDA label retrieved for Eteplirsen has no boxed warning; see the label's Warnings and Precautions for the recognised risks.

Do FAERS signals prove that Eteplirsen causes these side effects?

No. A disproportionality signal (PRR, ROR) means a reaction is reported more often with this drug than with others in the database. That can reflect a real effect, but also the disease being treated, stimulated reporting after news or litigation, co-medications, or how reports are coded. Causality needs temporality, dose-response, biological plausibility, consistency across data sources and, ideally, trial or cohort evidence. This page screens each term against the FDA label and ClinicalTrials.gov results; it does not establish causation.

Sources & methods

FDA label via openFDA (set ID 33bff678-7829-479e-9110-b8e33a0bc0aa, effective 20250813; matched on openfda.generic_name.exact:"ETEPLIRSEN")
Bayesian shrinkage measures computed offline over all 1,317,300 cached pairs: BCPNN information component (IC, with Norén closed-form IC025) and MGPS empirical-Bayes geometric mean (EBGM, EB05) with a two-component gamma prior fitted by maximum marginal likelihood (α₁=0.57, β₁=0.02, α₂=1.42, β₂=0.70, w=0.17); unstratified
FAERS pair statistics from the OpenPV build of 2026-09-14 (openFDA count= aggregation, field patient.drug.openfda.generic_name.exact, 27 reaction terms, min 3 co-reports); PRR/ROR with 95% CI, chi-square with Yates correction
Serious-only and stratified counts (serious:1, patient.patientsex, primarysource.qualification) from openFDA count queries; serious-only PRR uses the drug serious report total and the term global serious total (N_serious=11,882,979); strata shown only when n ≥ 20
Seriousness and sex counts from openFDA count=serious, patient.patientsex, seriousnessdeath

Page generated 2026-10-08. Label and PubChem text are retrieved automatically and may lag the current label; always check the current prescribing information. OpenPV is a beta research tool of the Open Source Medicine Foundation.