Elevidys is a recombinant gene therapy for Duchenne muscular dystrophy (DMD). Elevidys makes it possible for the body to make a protein (Elevidys micro-dystrophin) that contains copies of important sections of dystrophin proteins that were defective in the muscles of patients with Duchenne muscular dystrophy.

Elevidy is given as a single intravenous dose to Duchenne muscular dystrophy patients that are 4 to 5 years old and do not have contraindications to treatment.

Elevidys (delandistrogene moxeparvovec-rokl) received FDA approval on June 22, 2023, through the Accelerated Approval Pathway.

Acute Serious Liver Injury has been observed when using Elevidys. Before using this medicine liver function test should be performed, then monitor liver function (clinical exam, GGT, and total bilirubin) weekly for the first 3 months following Elevidys infusion.

Immune-mediated Myositis (an immune response affecting muscles).  Patients should contact their healthcare provider immediately if they experience any unexplained increased muscle pain, tenderness, or weakness, including swallowing difficulty, difficulty breathing or a weak voice, as these may be symptoms of myositis.

Myocarditis Acute serious myocarditis (inflammation of the heart) and troponin-I elevations have been observed following Elevidys infusion in clinical trials. Patients should contact their healthcare provider immediately if they experience chest pain and/or shortness of breath. 

While taking corticosteroids, an infection before or after the Elevidys infusion could lead to more serious complications. Contact a healthcare provider immediately if symptoms suggestive of infection are observed (e.g., coughing, wheezing, sneezing, runny nose, sore throat, or fever). 

• Elevidys is a single-dose (one-time treatment) given as a intravenous infusion.
• Before the Elevidys infusion, you will have your liver function assessed and other blood tests.
• You should not have an infusion while you have an infection.
• To reduce the risk associated with an immune response, you may be prescribed corticosteroids starting one day before the infusion.

The dose of Elevidys is based on body weight.
The recommended dose is 1.33 × 1014 vector genomes per kilogram (vg/kg) of body weight (or 10 mL/kg body weight).

The most common Elevidys side effects in clinical studies were: 

• vomiting
• nausea
• liver function test increased
• pyrexia
• thrombocytopenia.

The following are clinically significant adverse reactions

• acute serious liver injury
• immune-mediated myositis
• myocarditis.

Before starting the corticosteroid regimen required before Elevidys infusion, consider the patient’s vaccination status. Patients should, if possible, be brought up-to-date with all immunizations in current immunization guidelines. Vaccinations should be completed at least 4 weeks before starting the corticosteroid regimen.