What the label lists and what reports show
Delandistrogene moxeparvovec-rokl side effects
Delandistrogene moxeparvovec-rokl's FDA label: “Most common adverse reactions (incidence ≥5%) were vomiting and nausea, liver injury, pyrexia, thrombocytopenia, and troponin-I increased.” FAERS holds 342 reports naming it through July 30, 2026; the reaction reported most often, leaving out the conditions it is used for, product-use terms and outcomes, is vomiting (63 reports).
- No generic listed
- Boxed warning
Check it at the source: the delandistrogene moxeparvovec-rokl FDA label on DailyMed (effective August 12, 2026); the FDA FAERS public dashboard.
What does the patient leaflet say about side effects?
From the patient leaflet: What are the possible side effects of ELEVIDYS?ELEVIDYS can cause serious side effects. See “ What is the most important information I should know about ELEVIDYS? ” The most common side effects in individuals treated with ELEVIDYS include: vomiting nausea liver injury fever lower number of platelets, which is a kind of blood cell that helps you stop bleeding higher levels of heart protein that can detect damage to muscle cells in the heart (troponin-I) Tell your/your child's doctor about any side effects that bother you/your child or that do not go away. These are not all the possible side effects of ELEVIDYS. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics, Inc. at 1-888-727-3782.
Source: FDA drug label, Sarepta Therapeutics, Inc., effective August 12, 2026 (SPL set 3525abcd-fbd2-46c5-9706-cffdf2e8a361), via openFDA; the full label on DailyMed
What does the label list?
From the label: adverse reactionsMost common adverse reactions (incidence ≥5%) were vomiting and nausea, liver injury, pyrexia, thrombocytopenia, and troponin-I increased.
Table 3. Adverse reactions (Incidence ≥5%) following treatment with ELEVIDYS in Clinical Studies
| Adverse reactions | ELEVIDYS (N=156) % a Includes: AST increased, ALT increased, GGT increased, GLDH increased, GLDH level… b Includes: Thrombocytopenia, Platelet count decreased c Transient, mild, asymptomatic decrease in platelet counts d Includes: Troponin I increased, Troponin increased, Troponin I abnormal |
|---|---|
| Vomiting | 65 |
| Nausea | 43 |
| Liver injury a | 40 |
| Pyrexia | 28 |
| Thrombocytopenia b c | 8 |
| Troponin-I increased d | 8 |
Rows and numbers as the label prints them, under its own column headings (the first rows of a longer table where it runs on). A reaction in a trial table is not necessarily caused by the medicine, and trials differ from everyday use.
From the label: adverse reactions, continuedMost common adverse reactions (incidence ≥5%) were vomiting and nausea, liver injury, pyrexia, thrombocytopenia, and troponin-I increased. ( 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 the 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. The safety data described in this section reflect exposure to a one-time intravenous infusion of ELEVIDYS in 156 male patients with a confirmed mutation of the DMD gene in four clinical studies, including one completed open-label study, one ongoing open-label study, and two studies that included a double-blind, placebo-controlled period. Prior to ELEVIDYS infusion, patients in the ELEVIDYS treatment group had a mean age of 6.7 years (range: 3 to 20) and mean weight of 24.6 kg (range: 12.5 to 80.1). 144 patients received the recommended dose of 1.33 × 10 14 vg/kg, and 12 received a lower dose. Table 3 below presents adverse reactions from these four clinical studies. The most common adverse reactions (incidence ≥5%) across all studies are summarized in Table 3 .
Source: FDA drug label, Sarepta Therapeutics, Inc., effective August 12, 2026 (SPL set 3525abcd-fbd2-46c5-9706-cffdf2e8a361), via openFDA; the full label on DailyMed
What do FAERS reports show?
342 reports list delandistrogene moxeparvovec-rokl among the medicines taken, each a report of something that happened, not proof the medicine caused it; 7 are coded as a death and 160 as a hospital stay. Counts follow how widely a medicine is used and watched, and they are not a measure of how risky it is. Of the 23 reactions below, 8 are named by the same term (in US spelling) in the label's adverse reactions, warnings or patient leaflet; a reaction the label does not name may still be there under another word, and a report does not show that the medicine caused it.
The 25 most reported reactions
Source: FDA Adverse Event Reporting System (FAERS) via openFDA, reports through July 30, 2026; reactions as MedDRA preferred terms
What next?
- Delandistrogene moxeparvovec-rokl: the full record
- label, generics, price, shortages, recalls
- Report a side effect to the FDA
- MedWatch
- Side effects hub
- the most reported medicines and reactions