Delandistrogene Moxeparvovec-Rokl side effects: what 341 FDA adverse event reports show

Delandistrogene Moxeparvovec-Rokl · Elevidys · 10 reaction terms · 217 drug–reaction co-reports · data 2026-09-14

Drug class

Adeno-associated virus vector-based gene therapy

FDA label, Indications wording

Brand names

Elevidys

openFDA label

Who reports

341 reports · 61% serious · 7 with death outcome · 1% female

openFDA FAERS counts, all years, deduplication not applied

Used for

ELEVIDYS is indicated for the treatment of patients 4 years of age and older with Duchenne muscular dystrophy (DMD), who are ambulatory and have a confirmed mutation in the DMD gene . ELEVID.

FDA label, Indications and Usage

What it is

ELEVIDYS (delandistrogene moxeparvovec-rokl) is a recombinant gene therapy designed to deliver the gene encoding the ELEVIDYS micro-dystrophin protein. ELEVIDYS is a non-replicating, recombina.

FDA label, Description section

Boxed warning

ACUTE SERIOUS LIVER INJURY AND ACUTE LIVER FAILURE Acute serious liver injury, including life-threatening and fatal acute liver failure, has occurred with ELEVIDYS [see Warnings and Precautions ( 5.1 )]. Patients with preexisting liver impairment may be at higher risk [see Warnings and Precautions ( 5.1 )].

FDA 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 10 strongest signals

Labeled — boxed warning 3Labeled adverse reaction 1Labeled (related wording) 2Reporting / coding artifact 1Lab value / disease marker 2Not in label — unassessed 1

What the badges mean

Labeled — boxed warning The term (or its clinical synonym) appears in the FDA boxed warning: causality is regulator-recognised and considered serious.
Labeled adverse reaction The exact term appears in the label's warnings or adverse-reactions sections: a known, expected effect. The FAERS signal confirms the label rather than discovering anything.
Labeled (related wording) The label describes this effect under different wording (e.g. 'neutropenia' for 'neutrophil count decreased'). Treated as known.
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.
Lab value / disease marker A laboratory result, investigation or disease-activity marker (antibody positive, CRP abnormal, swollen joint count) not described in the label. These usually mirror the monitored disease or testing protocol rather than a drug effect; not screened further.
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. 0 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
Gamma-glutamyltransferase increased	43	119.1 90.0–157.5ROR 136.1 · χ² 4,913 / p <1e-300	113.0 EB05 87.4 · IC025 +5.2 3/3 methods agree	Lab value / disease marker	serious PRR 73.3	no trial data cached
Liver injury	29	73.6 52.0–104.3ROR 80.3 · χ² 2,005 / p <1e-300	70.3 EB05 51.3 · IC025 +4.4 3/3 methods agree	Labeled — boxed warning exact term	serious PRR 71.8	no trial data cached
Blood creatine phosphokinase increased	25	52.4 36.0–76.5ROR 56.5 · χ² 1,211 / p 1e-265	50.5 EB05 36.0 · IC025 +4.0 3/3 methods agree	Lab value / disease marker	serious PRR 30.8	no trial data cached
Myocarditis	8	43.3 21.8–86.0ROR 44.4 · χ² 290 / p 3e-65	39.5 EB05 21.4 · IC025 +2.4 3/3 methods agree	Labeled — boxed warning exact term	serious PRR 40.9	no trial data cached
Aspartate aminotransferase increased	32	37.0 26.6–51.4ROR 40.7 · χ² 1,086 / p 2e-238	36.2 EB05 26.9 · IC025 +4.0 3/3 methods agree	Labeled (related wording) related wording	serious PRR 28.7	no trial data cached
Transaminases increased	13	35.9 21.1–61.2ROR 37.3 · χ² 407 / p 8e-91	34.2 EB05 21.3 · IC025 +3.0 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 27.7	no trial data cached
Alanine aminotransferase increased	35	35.0 25.5–47.8ROR 38.8 · χ² 1,123 / p 1e-246	34.3 EB05 25.8 · IC025 +4.0 3/3 methods agree	Labeled (related wording) related wording	serious PRR 28.1	no trial data cached
Drug-induced liver injury	17	33.8 21.3–53.7ROR 35.5 · χ² 509 / p 4e-113	32.7 EB05 21.6 · IC025 +3.3 3/3 methods agree	Not in label — unassessed	serious PRR 32.1	no trial data cached
Device occlusion	6	26.5 12.0–58.5ROR 26.9 · χ² 123 / p 8e-29	23.5 EB05 9.8 · IC025 +1.7 3/3 methods agree	Reporting / coding artifact	–	no trial data cached
Hepatotoxicity (liver damage)	9	24.9 13.1–47.5ROR 25.5 · χ² 183 / p 4e-42	23.4 EB05 12.6 · IC025 +2.3 3/3 methods agree	Labeled — boxed warning synonym match	serious PRR 23.9	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
Gamma-glutamyltransferase increased	43	119.1 90.0–157.5ROR 136.1 · χ² 4,913 / p <1e-300	113.0 EB05 87.4 · IC025 +5.2 3/3 methods agree	Lab value / disease marker	serious PRR 73.3	no trial data cached
Alanine aminotransferase increased	35	35.0 25.5–47.8ROR 38.8 · χ² 1,123 / p 1e-246	34.3 EB05 25.8 · IC025 +4.0 3/3 methods agree	Labeled (related wording) related wording	serious PRR 28.1	no trial data cached
Aspartate aminotransferase increased	32	37.0 26.6–51.4ROR 40.7 · χ² 1,086 / p 2e-238	36.2 EB05 26.9 · IC025 +4.0 3/3 methods agree	Labeled (related wording) related wording	serious PRR 28.7	no trial data cached
Liver injury	29	73.6 52.0–104.3ROR 80.3 · χ² 2,005 / p <1e-300	70.3 EB05 51.3 · IC025 +4.4 3/3 methods agree	Labeled — boxed warning exact term	serious PRR 71.8	no trial data cached
Blood creatine phosphokinase increased	25	52.4 36.0–76.5ROR 56.5 · χ² 1,211 / p 1e-265	50.5 EB05 36.0 · IC025 +4.0 3/3 methods agree	Lab value / disease marker	serious PRR 30.8	no trial data cached
Drug-induced liver injury	17	33.8 21.3–53.7ROR 35.5 · χ² 509 / p 4e-113	32.7 EB05 21.6 · IC025 +3.3 3/3 methods agree	Not in label — unassessed	serious PRR 32.1	no trial data cached
Transaminases increased	13	35.9 21.1–61.2ROR 37.3 · χ² 407 / p 8e-91	34.2 EB05 21.3 · IC025 +3.0 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 27.7	no trial data cached
Hepatotoxicity (liver damage)	9	24.9 13.1–47.5ROR 25.5 · χ² 183 / p 4e-42	23.4 EB05 12.6 · IC025 +2.3 3/3 methods agree	Labeled — boxed warning synonym match	serious PRR 23.9	no trial data cached
Myocarditis	8	43.3 21.8–86.0ROR 44.4 · χ² 290 / p 3e-65	39.5 EB05 21.4 · IC025 +2.4 3/3 methods agree	Labeled — boxed warning exact term	serious PRR 40.9	no trial data cached
Device occlusion	6	26.5 12.0–58.5ROR 26.9 · χ² 123 / p 8e-29	23.5 EB05 9.8 · IC025 +1.7 3/3 methods agree	Reporting / coding artifact	–	no trial data cached

Unlabeled candidate signals

Every screened signal with n ≥ 20 was either labeled, an indication term, or an artifact pattern — no unlabeled candidate stood out in the top signals.

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 Delandistrogene Moxeparvovec-Rokl?

In FDA FAERS, the most frequently co-reported reaction terms for Delandistrogene Moxeparvovec-Rokl are gamma-glutamyltransferase increased, alanine aminotransferase increased, aspartate aminotransferase increased, liver injury, blood creatine phosphokinase increased, drug-induced liver injury. 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 Delandistrogene Moxeparvovec-Rokl have an FDA boxed warning?

Yes. The current FDA label carries a boxed warning: ACUTE SERIOUS LIVER INJURY AND ACUTE LIVER FAILURE Acute serious liver injury, including life-threatening and fatal acute liver failure, has occurred with ELEVIDYS [see Warnings and Precautions ( 5.1 )]. Patients with preexisting liver impairment may be at higher risk [see Warnings and Precautions ( 5.1 )].

Do FAERS signals prove that Delandistrogene Moxeparvovec-Rokl 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 3525abcd-fbd2-46c5-9706-cffdf2e8a361, effective 20260812; matched on openfda.generic_name.exact:"DELANDISTROGENE MOXEPARVOVEC-ROKL")
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, 10 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.