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Results in ambulatory people


One of the key elements measured in ELEVIDYS clinical trials is whether the treatment worked. This included considering the amount of ELEVIDYS micro-dystrophin produced and whether there was a change in motor function. Here’s what we learned.


Impact on ELEVIDYS micro-dystrophin

Across all trials, participants produced an average of 34% to 51% ELEVIDYS micro-dystrophin 3 months after treatment

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STUDY 1

Part 1

43%

6 participants

STUDY 1

Part 2

41%

21 participants

STUDY 2

 

51%

40 participants

STUDY 3

Part 1

34%

17 participants

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In Study 3, since people receiving placebo (14 participants) did not receive ELEVIDYS, they had 0% ELEVIDYS micro-dystrophin

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HOW DID IT WORK?

Results were measured using a test called western blot, which looks at a small piece of muscle from a person’s body to see if micro-dystrophin was produced. The numbers reflect the amount produced, compared to the start of the trial, if 100% was the typical level in a person without Duchenne.

People with Duchenne who are untreated typically have little to no dystrophin.

HOW DID IT WORK?

Results were measured using a test called western blot, which looks at a small piece of muscle from a person’s body to see if micro-dystrophin was produced. The numbers reflect the amount produced, compared to the start of the trial, if 100% was the typical level in a person without Duchenne.

People with Duchenne who are untreated typically have little to no dystrophin.

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Impact on motor function
 

After 1 year, people in Study 3, Part 1, who were treated with ELEVIDYS scored an average of 0.7 points higher on the North Star Ambulatory Assessment (sometimes called NSAA or North Star) than those who received placebo. The difference was not statistically significant.

Average change in NSAA score after 1 year of treatment

ELEVIDYS effect on NSAA after 1 year
HOW DID IT WORK?

The NSAA is a broad measure used to monitor overall ambulatory function over time.

At the start of the trial, participants completed 17 different tasks to measure motor function, including activities like the ability to stand up from a chair, climb stairs, jump, and balance on 1 leg. For each task, participants received a score of 0 (unable), 1 (with help), or 2 (independently). Half the participants then received ELEVIDYS and the other half received placebo (no ELEVIDYS). One year later, everyone repeated the test to see if their results changed from the start of the trial.

HOW DID IT WORK?

The NSAA is a broad measure used to monitor overall ambulatory function over time.

At the start of the trial, participants completed 17 different tasks to measure motor function, including activities like the ability to stand up from a chair, climb stairs, jump, and balance on 1 leg. For each task, participants received a score of 0 (unable), 1 (with help), or 2 (independently). Half the participants then received ELEVIDYS and the other half received placebo (no ELEVIDYS). One year later, everyone repeated the test to see if their results changed from the start of the trial.

Similar results were seen in Study 1 when measured 48 weeks after treatment in people aged 4 to 7 years: those who received ELEVIDYS (20 participants) scored 0.8 points higher, on average, than those who received placebo (no ELEVIDYS, 21 participants). The difference was not statistically significant.

All participants were on stable doses of steroids at the start of the trial regardless of whether they received ELEVIDYS or placebo.

ELEVIDYS improved participants’ speed on the time to rise from floor test

Study 3, Part 1

Average change in time to rise from floor from start of trial to 1 year 
124 participants aged 4-7 years

Time to rise from floor test
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Participants who received ELEVIDYS (63 people)
0.27 seconds
faster
vs start of trial

Participants who received placebo (61 people)
0.37 seconds
slower
vs start of trial

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Those who received ELEVIDYS were able to get up from the floor

0.64 seconds faster

than those on placebo
(no ELEVIDYS) after 1 year (on average)

 

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HOW DID IT WORK?

At the start of the trial, participants were asked to stand up from lying on their back as quickly as they could, and these times were recorded. Half the participants then received ELEVIDYS and the other half received placebo (no ELEVIDYS). One year later, everyone repeated the test to see if their results changed from the start of the trial. While results varied across participants, the average difference is noted above.

HOW DID IT WORK?

At the start of the trial, participants were asked to stand up from lying on their back as quickly as they could, and these times were recorded. Half the participants then received ELEVIDYS and the other half received placebo (no ELEVIDYS). One year later, everyone repeated the test to see if their results changed from the start of the trial. While results varied across participants, the average difference is noted above.

All participants were on stable doses of steroids at the start of the trial regardless of whether they received ELEVIDYS or placebo.

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Participants treated with ELEVIDYS could walk/run faster on a test of motor function

Study 3, Part 1

Average change in 10-meter walk/run time from start of trial to 1 year 

124 participants aged 4-7 years

 

Time to walk or run 10-meters
The effect of ELEVIDYS and placebo on the time to walk or run 10 meters, Effect of ELEVIDYS on time to walk or run 10 meters compared to placebo
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Participants who received ELEVIDYS (63 people)
0.34 seconds
faster
vs start of trial

Participants who received placebo (61 people)
0.08 seconds
slower
vs start of trial

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Those who received ELEVIDYS were able to walk/run 10 meters

0.42 seconds faster

than those on placebo
(no ELEVIDYS) after 1 year (on average)

 

The effect of ELEVIDYS and placebo on the time to walk or run 10 meters, Effect of ELEVIDYS on time to walk or run 10 meters compared to placebo
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HOW DID IT WORK?

At the start of the trial, participants were asked to run or walk 10 meters (approximately 30 feet) as quickly as they could, and these times were recorded. Half the participants then received ELEVIDYS and the other half received placebo (no ELEVIDYS). One year later, everyone repeated the test to see if their results changed from the start of the trial. While results varied across participants, the average difference is noted above.

HOW DID IT WORK?

At the start of the trial, participants were asked to run or walk 10 meters (approximately 30 feet) as quickly as they could, and these times were recorded. Half the participants then received ELEVIDYS and the other half received placebo (no ELEVIDYS). One year later, everyone repeated the test to see if their results changed from the start of the trial. While results varied across participants, the average difference is noted above.

All participants were on stable doses of steroids at the start of the trial regardless of whether they received ELEVIDYS or placebo.

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Next up: Explore safety

Now that you’ve seen the impact ELEVIDYS had in the clinical trials, learn more about safety, including possible side effects.

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Boy who received treatment with ELEVIDYS

What is ELEVIDYS (delandistrogene moxeparvovec-rokl)?

ELEVIDYS is a prescription gene therapy used to treat ambulatory individuals at least 4 years old with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene.

ELEVIDYS is approved under accelerated approval for non-ambulatory patients at least 4 years old with DMD who have a confirmed mutation in the DMD gene. Accelerated approval allows for drugs to be approved based on a marker that is considered reasonably likely to predict a clinical benefit. ELEVIDYS treatment increased the marker, ELEVIDYS micro-dystrophin in skeletal muscle. Verification of a clinical benefit may be needed for ELEVIDYS to continue to be approved for non-ambulatory patients with DMD.

Important Safety Information

Who should not receive ELEVIDYS?
Individuals with certain types of mutations, any deletion in exon 8 and/or exon 9 in the DMD gene, should not receive ELEVIDYS.

What is the most important information to know about ELEVIDYS?
Infusion-related reactions, including hypersensitivity and serious allergic reactions (anaphylaxis), have occurred during and after ELEVIDYS infusion. Symptoms may include fast heart rate, fast breathing, swollen lips, shortness of breath, nostrils widening, hives, red and blotchy skin, itchy or inflamed lips, rash, vomiting, nausea, chills, and fever. Your doctor will monitor you during and at least 3 hours after ELEVIDYS infusion. If an infusion-related reaction occurs, your doctor may slow or stop the ELEVIDYS infusion and provide additional medical treatment as needed. Contact a healthcare provider immediately if infusion-related symptoms occur. 

ELEVIDYS can increase certain liver enzyme levels and cause acute serious liver injury. Patients will receive oral corticosteroid medication before and after infusion with ELEVIDYS and will undergo weekly blood tests to monitor liver enzyme levels for 3 months after treatment. Contact a healthcare provider immediately if the patient’s skin and/or whites of the eyes appear yellowish or if the patient misses a dose of corticosteroid or vomits it up. 

Administration of ELEVIDYS may be delayed in patients who have acute liver disease until the condition is resolved or under control. Patients with preexisting liver impairment, chronic liver infection, or acute liver disease may be at higher risk of acute serious liver injury.

Immune-mediated myositis (an immune response affecting muscles) was observed in patients with a deletion mutation in the DMD gene that is contraindicated. Patients with certain mutation deletions (in exons 1 to 17 and/or exons 59 to 71) may be at risk for a severe immune-mediated myositis reaction. Caregivers should contact a healthcare provider immediately if the patient experiences any unexplained increased muscle pain, tenderness, or weakness, including difficulty swallowing, breathing, or speaking, as these may be symptoms of myositis. 

Myocarditis (inflammation of the heart) has been observed within days following ELEVIDYS infusion. The patient’s doctor will conduct weekly blood tests for the first month after treatment to evaluate troponin-I (a cardiac protein that can detect damage to muscle cells in the heart). Caregivers should contact a healthcare provider immediately if the patient begins to experience chest pain and/or shortness of breath. More frequent monitoring may be required if the patient has cardiac symptoms. 

Patients need to have blood tests to ensure that they do not have antibodies that may prevent them from being able to receive ELEVIDYS, as introducing the gene therapy could increase the risk of a severe allergic reaction or prevent desired therapeutic levels. Treatment with ELEVIDYS is not recommended for patients who have high antibodies to the vector, the part of gene therapy used to deliver ELEVIDYS. 

Due to the need to follow a corticosteroid regimen, an infection (such as cold, flu, gastroenteritis [stomach flu], otitis media [ear infection], bronchiolitis [respiratory infection], etc) before or after ELEVIDYS infusion could lead to more serious complications. Caregivers should contact a healthcare provider immediately if they see any symptoms suggestive of infection, such as coughing, wheezing, sneezing, runny nose, sore throat, or fever.

Are there any considerations for vaccination schedules and ELEVIDYS?
Patient vaccinations should be up to date with current immunization guidelines. Vaccinations should be received at least 4 weeks prior to starting the corticosteroid regimen that is required before receiving ELEVIDYS.

Are there any precautions that need to be considered when handling a patient’s bodily waste?
Vector shedding of ELEVIDYS occurs primarily through body waste. Patients and caregivers should use proper hand hygiene, such as hand washing when coming into direct contact with patient body waste. Place potentially contaminated materials that may have the patient’s bodily fluids/waste in a sealable bag and dispose into regular trash. Precautions should be followed for 1 month after ELEVIDYS infusion.

What are the possible or likely side effects of ELEVIDYS?
The most common side effects that occurred in patients treated with ELEVIDYS were vomiting, nausea, liver injury, fever, and decreased platelet counts. 

The safety information provided here is not comprehensive. Talk to the patient’s doctor about any side effects that bother the patient or that don’t go away.

You are encouraged to 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 for ELEVIDYS.

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