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Required monitoring1

Weekly safety monitoring is required for at least 3 months after ELEVIDYS infusion (or longer, if clinically indicated). Use the below schedule, which was informed by FDA guidance and leading medical experts, as a guide.

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Safety considerations

Adverse reactions may occur at any time, but below is when they were typically seen:

  • Within 2 weeks of infusion: nausea, vomiting (as early as on infusion day), thrombocytopenia, pyrexia
  • Within 1 month: myocarditis, increased troponin-I levels
  • Within 2 months: immune-mediated myositis, liver injury

See Important Safety Information for additional safety considerations when infusing ELEVIDYS.

Postinfusion assessments are required for patient safety

Liver function monitoring

Liver function

  • Acute serious liver injury, including life-threatening and fatal acute liver failure, has occurred with ELEVIDYS. Administration of ELEVIDYS may result in elevations of liver enzymes (eg, GGT, ALT, AST) and total bilirubin, typically seen within 8 weeks
  • Patients with preexisting liver impairment, chronic hepatic condition, or acute liver disease (eg, acute hepatic viral infection) may be at higher risk of acute serious liver injury or acute liver failure
  • Monitor liver function weekly for the first 3 months via clinical exam and analysis of AST, ALT, GGT, albumin, aPTT, INR, and total bilirubin levels. Continue monitoring if clinically indicated, until results are unremarkable (normal clinical exam and GGT and total bilirubin levels return to near baseline levels)
  • Adjust the postinfusion oral corticosteroid regimen as needed for patients with liver function abnormalities, using the chart below for guidance
  • Obtain prompt consultation with a specialist (eg, gastroenterologist or hepatologist) if acute serious liver injury or impending acute liver failure is suspected
Platelet count monitoring

Platelet count

  • Transient, mild, asymptomatic decrease in platelet count has been observed following ELEVIDYS administration
  • Monitor platelet counts weekly for the first 2 weeks, and continue if clinically indicated
Myocarditis monitoring

Myocarditis

  • Acute, serious, life-threatening myocarditis and troponin-I elevations have been observed within 24 hours to more than 1 year following ELEVIDYS infusion
  • If a patient experiences myocarditis, those with preexisting left ventricle ejection fraction (LVEF) impairment may be at higher risk of adverse outcomes. Patients with moderate to severe LVEF impairment have not been studied in clinical trials with ELEVIDYS
  • Monitor troponin-I weekly for the first month. More frequent or longer monitoring may be required in the presence of cardiac symptoms, such as chest pain or shortness of breath, if clinically indicated

ALT=alanine aminotransferase; aPTT=activated partial thromboplastin time; AST=aspartate aminotransferase; GGT=gamma-glutamyl transferase; INR=international normalized ratio.

Continuing the required corticosteroid regimen

Immune responses to the AAVrh74 vector can occur after administration of ELEVIDYS. To reduce the risk associated with an immune response, patients must continue treatment with systemic oral corticosteroids for at least 60 days after the infusion, unless earlier tapering is clinically indicated.

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Recommended oral corticosteroid regimen dose modification for liver function abnormalities following ELEVIDYS infusion*

Peri-ELEVIDYS infusion corticosteroid dosing

Modified oral corticosteroid dose following ELEVIDYS infusion (prednisone equivalent)†‡

Recommended maximum total daily oral dose (prednisone equivalent)†‡

Baseline + 1 mg/kg/day

Increase to 2 mg/kg/day (and continue baseline dose)

120 mg/day

Baseline + 1 mg/kg/day taken on days without high-dose corticosteroid treatment

Increase to 2 mg/kg/day taken on days without high-dose corticosteroid treatment (and continue baseline dose)

120 mg/day

1.5 mg/kg/day

Increase from 1.5 mg/kg/day to 2.5 mg/kg/day

120 mg/day

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* GGT ≥3 times baseline and/or other clinically significant liver function abnormalities (eg, total bilirubin > ULN).

† For GGT or bilirubin elevations that do not respond to these oral corticosteroid increases, IV bolus corticosteroids may be considered.

‡ Corticosteroids other than prednisone and prednisolone have not been studied for use as a peri-ELEVIDYS infusion corticosteroid.

ULN=upper limit of normal.


Consider IV bolus corticosteroids instead of oral corticosteroids for GGT or bilirubin elevations that do not respond after 1 week of increased oral corticosteroids. Consult with a specialist experienced in immunosuppressive therapy for additional interventions, as needed.

It is critical that patients adhere to the required pre- and postinfusion oral corticosteroid regimen to reduce the risk of an adverse immune response

Max received treatment with ELEVIDYS
Austin received treatment with ELEVIDYS

Caregiver counseling information

Provide a copy of the FDA-approved Medication Guide to the caregiver, review the contents with them, and advise them to read it in its entirety. Counsel caregivers on what to expect postinfusion and reiterate the importance of compliance with the required corticosteroids and monitoring schedule.

  1. Speak with caregivers about the risks of ELEVIDYS treatment, and advise them to contact you immediately if the patient:  

    • Has yellowish skin and/or whites of their eyes, misses a dose of corticosteroid or vomits it up, or experiences a change in mental status. These could be indicators of increased liver enzymes, which can lead to acute serious liver injury, acute liver failure, or death.
    • Shows signs suggestive of an infection (coughing, wheezing, sneezing, runny nose, sore throat, fever), as these can lead to more serious complications, including death.
    • Begins to experience chest pain and/or shortness of breath, as these could be signs of myocarditis (inflammation of the heart)
    • Experiences a fast heart rate, fast breathing, swollen lips, being short of breath, nostrils widening, hives, red and blotchy skin, itchy or inflamed lips, rash, vomiting, nausea, chills, or fever. These could be signs of an infusion-related reaction

    • Has any unexplained increased muscle pain, tenderness, or weakness, including difficulty swallowing, difficulty breathing, or difficulty speaking, as these may be symptoms of myositis

  2. Remind caregivers about the importance of the oral treatment-related corticosteroids, why it’s critical to follow the prescribed dose both before and after treatment, and how they can help reduce the risk of an immune response to the ELEVIDYS vector (AAVrh74)

  3. Reinforce the importance of the weekly postinfusion monitoring of liver function, troponin-I levels, and platelet counts. Alert caregivers that they will need to remain close to an appropriate healthcare facility, as determined by the provider, for at least 2 months following treatment

  4. Advise caregivers that an infection (eg, cold, flu, gastroenteritis, otitis media, bronchiolitis, or pneumonia) before or after infusion could lead to more serious complications.

  5. Remind patients, caregivers, and family members about proper hand hygiene for 1 month following infusion. Advise them to wash their hands when they come in contact with the patient’s body waste, and to place potentially contaminated materials that may have the patient’s bodily fluids/waste in a sealable bag in the regular trash

     

References:

  • 1. ELEVIDYS. Prescribing information. Sarepta Therapeutics, Inc; November 2025.

Indication

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

Limitations of Use
ELEVIDYS is not recommended in patients with:

  • Preexisting liver impairment (defined as gamma-glutamyl transferase [GGT] > 2 x upper limit of normal or total bilirubin > the upper limit of normal not due to Gilbert's syndrome) or active hepatic viral infection due to the high risk of acute serious liver injury and acute liver failure.
  • Recent vaccination (within 4 weeks of treatment) due to immunogenicity and potential safety concerns.
  • Active or recent (within 4 weeks) infections due to safety concerns.

Please see full Prescribing Information, including Boxed Warning, and Medication Guide.

Important Safety Information, including Boxed Warning

BOXED WARNING: Acute Serious Liver Injury and Acute Liver Failure

Acute serious liver injury, including life-threatening and fatal acute liver failure, has occurred.
Patients with preexisting liver impairment may be at higher risk. 

Prior to infusion, assess liver function by clinical examination and laboratory testing. Administer systemic corticosteroids before and after ELEVIDYS infusion. Continue to monitor liver function weekly for the first 3 months after infusion and continue until results are unremarkable. 

Instruct patients to maintain proximity to an appropriate healthcare facility, as determined by the healthcare provider, for at least 2 months following ELEVIDYS infusion.

Obtain prompt consultation with a specialist (e.g., gastroenterologist or hepatologist) if acute serious liver injury or impending acute liver failure is suspected.

CONTRAINDICATION
ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9, including a deletion of any portion or the entirety of these exons, in the DMD gene. 

WARNINGS AND PRECAUTIONS
Acute Serious Liver Injury and Acute Liver Failure
See Boxed Warning.

  • Acute serious liver injury marked by elevations of liver enzymes (e.g., GGT, ALT) and total bilirubin and acute liver failure has occurred with ELEVIDYS. Onset of the liver injury typically begins within 8 weeks of ELEVIDYS administration. In non-ambulatory patients treated with ELEVIDYS, acute liver failure with fatal outcome has occurred in the clinical and post-marketing settings.
  • Life-threatening mesenteric vein thrombosis, complicated by bowel ischemia and necrosis, and portal hypertension have been reported following acute liver injury associated with ELEVIDYS in a non-ambulatory patient.
  • Patients with preexisting liver impairment, chronic hepatic condition, or acute liver disease (e.g., acute hepatic viral infection) may be at higher risk of acute serious liver injury or acute liver failure. Postpone ELEVIDYS administration in patients with acute liver disease until resolved or controlled.
  • Systemic corticosteroid treatment is recommended for patients before and after ELEVIDYS infusion. Adjust corticosteroid regimen when indicated.

Serious infections

  • Increased susceptibility to serious infections may occur due to concomitant administration of corticosteroid regimen and additional immunosuppressants, and ELEVIDYS. Serious respiratory infections, including with fatal outcomes, have occurred in patients taking immunosuppressant corticosteroids required for ELEVIDYS administration.
  • Monitor patients for signs and symptoms of infection before and after ELEVIDYS administration and treat appropriately.
  • Administer immunizations according to best clinical practices and immunization guidelines prior to initiation of the corticosteroid regimen required before ELEVIDYS infusion.
  • Avoid administration of ELEVIDYS to patients with active infections.

Myocarditis

  • Acute, serious, life-threatening myocarditis and troponin-I elevations have been observed within 24 hours to more than 1 year following ELEVIDYS infusion.
  • If a patient experiences myocarditis, those with pre-existing left ventricle ejection fraction (LVEF) impairment may be at higher risk of adverse outcomes.
  • Monitor troponin-I before ELEVIDYS infusion and weekly for the first month following infusion and continue monitoring if clinically indicated, until results return to near baseline levels or stabilize.
  • More frequent monitoring may be warranted in the presence of cardiac symptoms, such as chest pain or shortness of breath.
  • Advise patients to contact a physician immediately if they experience cardiac symptoms.

Infusion-related Reactions

  • Infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred during or up to several hours following ELEVIDYS administration. Closely monitor patients during and for at least 3 hours after the end of infusion. If symptoms of infusion-related reactions occur, slow or stop the infusion and give appropriate treatment. Once symptoms resolve, the infusion may be restarted at a lower rate.
  • ELEVIDYS should be administered in a setting where treatment for infusion-related reactions is immediately available.
  • Discontinue infusion for anaphylaxis. 

Immune-mediated Myositis

  • Immune-mediated myositis, including serious and life-threatening events, has occurred approximately 1 month following ELEVIDYS infusion. Signs and symptoms include severe muscle weakness, including dysphagia, dyspnea, dysphonia, and hypophonia.
  • Severe to life-threatening immune-mediated myositis has been reported in patients with deletions including portions of exons 1-17 and/or exons 59-71 of the DMD gene.
  • Regardless of genetic mutation, advise patients to contact a physician immediately if they experience any unexplained increased muscle pain, tenderness, or weakness, including dysphagia, dyspnea, dysphonia, or hypophonia, as these may be symptoms of myositis. Consider additional immunomodulatory treatment based on patient's clinical presentation and medical history if these symptoms occur.

Preexisting Immunity against AAVrh74

  • In AAV-vector based gene therapies, preexisting anti-AAV antibodies may impede transgene expression at desired therapeutic levels. Following treatment with ELEVIDYS, all patients developed anti-AAVrh74 antibodies.
  • Perform baseline testing for the presence of anti-AAVrh74 total binding antibodies prior to ELEVIDYS administration.
  • ELEVIDYS administration is not recommended in patients with elevated anti-AAVrh74 total binding antibody titers ≥1:400.

ADVERSE REACTIONS

  • The most common adverse reactions (incidence ≥5%) reported in clinical studies were vomiting, nausea, liver injury, pyrexia, thrombocytopenia, and troponin-I increased.

Report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA (1-888-727-3782).

Please see full Prescribing Information, including Boxed Warning, and Medication Guide.