How much does Elevidys cost?

Elevidys costs $3.2 million for a one-time, single-dose intravenous (IV) infusion, making it one of the most expensive drugs in the world. Elevidys was the first gene therapy approved for the treatment of Duchenne muscular dystrophy (DMD).

Elevidys (delandistrogene moxeparvovec-rokl) was initially approved by the FDA on June 22, 2023 and is manufactured by Sarepta Therapeutics.

Does Elevidys have a copay assistance program?

Sarepta Therapeutics offers a copay assistance program for patients with commercial (private) health insurance in the U.S. This copay program may help with some out-of-pocket insurance costs related to receiving Elevidys therapy, such as copays, coinsurance, and deductibles.

Examples of commercial insurance in the U.S. include those provided from your employer or that you may have bought yourself through the Healthcare.gov Marketplace.

The Sarepta copay assistance program is not available for people with government-sponsored insurance like Medicare, Medicaid or TriCare.

How can I get Elevidys for my child?

Your healthcare provider is your main resource for discussing your child’s medical needs. Once you and your doctor decide that Elevidys is the right treatment path, a Sarepta Case Manager will help to facilitate your next steps.

You and your doctor will need to fill out specific forms from Sarepta that allow Case Managers to investigate your insurance benefits, financial assistance needs and to provide genetic testing information.

SareptAssist, offered by Sarepta Therapeutics is a customized and personalized, one-on-one support program to help guide you and your child on the Elevidys treatment journey. This program can help you:

• Understand treatment requirements and test
• Prepare for and understand the treatment process and logistic
• Learn about financial assistance options and insurance benefit
• Locate helpful resources for rare diseases like DMD, including independent charitable organization
• Provide education and support over the long-term

To learn more, call 1-888-SAREPTA (1-888-727-3782), Monday through Friday, 8:30 am 6:30 pm ET to talk to a SareptAssist Case Manager or go to SareptaAssist.com.

What is Elevidys used to treat?

Elevidys is indicated for the treatment of Duchenne Muscular Dystrophy (DMD) in people at least 4 years of age who are either:

• Ambulatory and have a confirmed mutation in the DMD gene.
• Non-ambulatory and have a confirmed mutation in the DMD gene. The DMD indication in non-ambulatory patients is approved under an FDA-accelerated approval.
• Elevidys cannot be used in people with any deletion in exon 8 and/or exon 9 in the DMD gene.

Accelerated approval in non-ambulatory patients was based on expression of Elevidys micro-dystrophin observed in patients treated with Elevidys. Continued approval for this use may depend upon further clinical trial results.

What causes Duchenne Muscular Dystrophy (DMD)?

Duchenne Muscular Dystrophy (DMD) is an inherited, genetic disease caused by a mutation in the dystrophin gene that results in low levels of the dystrophin protein needed to strengthen muscle fibers.

DMD is a degenerative disease that results in muscle weakness and loss of muscle tissue which worsens over time. DMD primarily affects males starting in early childhood, often between the ages of 2 and 3. It is the most common childhood form of muscular dystrophy.

In addition to progressive weakness and loss of skeletal and heart muscles, it can lead to premature death in the second or third decade of life due to heart problems and lung failure. The average life expectancy of people with DMD is about 27 years.

How does Elevidys work in DMD?

Elevidys is an adeno-associated virus vector-based gene therapy and was designed to treat the underlying cause of Duchenne Muscular Dystrophy (DMD). It works by delivering a gene into the muscle tissue that codes for a functional shortened dystrophin (called Elevidys micro-dystrophin). It was the first gene therapy approved for DMD.

Elevidys is given as an intravenous (IV) infusion over 1 to 2 hours, but your treatment visit will last longer, as determined by your doctor. Your doctor may also prescribe corticosteroids medicines starting one day before the infusion to help reduce the risk associated with an immune response reaction.

Learn more: Dosing of Elevidys (in more detail)

Safety

Warnings and precautions for Elevidys treatment include acute serious liver injury, immune-mediated myositis (muscle inflammation) and myocarditis (heart muscle inflammation).

Contact your doctor right away if:

• Your child’s skin and/or whites of the eyes appear a yellowish color, as this may be a sign of increased liver enzymes and liver injury.
• Your child has unexplained increased muscle pain, tenderness, or weakness, including trouble swallowing, breathing, or speaking, as these may be a sign of an immune response affecting muscles (immune-mediated myositis)
• Your child experiences chest pain and/or shortness of breath, as these may be a sign of inflammation of the heart muscles (myocarditis).

Common side effects that occurred in at least 5% of patients in studies include nausea and vomiting, liver injury, pyrexia (fever), and thrombocytopenia (low platelet counts).

Related: What are the new drugs for Duchenne Muscular Dystrophy (DMD)?

This is not all the information you need to know about Elevidys (delandistrogene moxeparvovec-rokl) for safe and effective use and does not take the place of talking to your doctor about your treatment. Review the full product information and discuss any questions you have with your doctor or other health care provider.