What the label lists and what reports show
Golodirsen side effects
Golodirsen's FDA label: “The most common adverse reactions (incidence ≥20% and higher than placebo) were headache, pyrexia, fall, abdominal pain, nasopharyngitis, cough, vomiting, and nausea.” FAERS holds 435 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 poor venous access (63 reports).
- No generic listed
Check it at the source: the golodirsen FDA label on DailyMed (effective March 13, 2025, Sarepta Therapeutics, Inc.); the FDA FAERS public dashboard.
What does the label list?
From the label: adverse reactionsThe most common adverse reactions (incidence ≥20% and higher than placebo) were headache, pyrexia, fall, abdominal pain, nasopharyngitis, cough, vomiting, and nausea.
Table 1: Adverse Reactions That Occurred in At Least 20% of VYONDYS 53-Treated Patients and at a Rate Greater than Placebo in Studies 1 and 2
| Adverse Reaction | VYONDYS 53 (N = 41) % | Placebo (N = 21) % |
|---|---|---|
| Headache | 41 | 10 |
| Pyrexia | 41 | 14 |
| Fall | 29 | 19 |
| Abdominal pain | 27 | 10 |
| Nasopharyngitis | 27 | 14 |
| Cough | 27 | 19 |
| Vomiting | 27 | 19 |
| Nausea | 20 | 10 |
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, continuedThe following serious adverse reactions are described below and elsewhere in the labeling: Hypersensitivity Reactions [see Warnings and Precautions ( 5.1 )] The most common adverse reactions (incidence ≥20% and higher than placebo) were headache, pyrexia, fall, abdominal pain, nasopharyngitis, cough, vomiting, and nausea. ( 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. In the VYONDYS 53 clinical development program, 58 patients received at least one intravenous dose of VYONDYS 53, ranging between 4 mg/kg (0.13 times the recommended dosage) and 30 mg/kg (the recommended dosage). All patients were male and had genetically confirmed Duchenne muscular dystrophy. Age at study entry was 6 to 13 years. Most (86%) patients were Caucasian. VYONDYS 53 was studied in 2 double-blind, placebo-controlled studies. …
Source: FDA drug label, Sarepta Therapeutics, Inc., effective March 13, 2025 (SPL set 35c227d1-5b24-44b0-b5d3-f0f6b1c46bd5), via openFDA; the full label on DailyMed
What do FAERS reports show?
435 reports list golodirsen among the medicines taken, each a report of something that happened, not proof the medicine caused it; 7 are coded as a death and 67 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 21 reactions below, 9 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?
- Golodirsen: 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