If you’re looking at treatment options for spinal muscular atrophy (SMA), you may want to learn more about Zolgensma. It’s a prescription drug used to treat SMA in children under 2 years of age. For this use, the drug is prescribed for children with SMA caused by changes in the SMN1 gene.

Zolgensma contains the active ingredient onasemnogene abeparvovec-xioi. (An active ingredient is what makes a drug work.) It’s a kind of gene therapy that replaces the abnormal SMN1 gene with a normal version.

Zolgensma comes as a liquid suspension. It’s given as an intravenous (IV) infusion (an injection into a vein given over time) by a healthcare professional.

Keep reading for details on Zolgensma and cost and how to save money on prescriptions.

Note: For more details on Zolgensma, see this in-depth article.

The price you pay for Zolgensma can vary. Your cost may depend on the treatment plan prescribed by your child’s doctor and your insurance coverage (if you have it). It will also depend on how much you have to pay for a visit to a treatment center for your child to receive Zolgensma.

To find out how much you’ll pay for Zolgensma, talk with your doctor or insurance provider.

Below are answers to some frequently asked questions about Zolgensma and cost.

What is Zolgensma’s cost in the U.S.A.?

Zolgensma’s cost in the United States can vary from person to person. This is because the cost is affected by several factors, including the treatment plan prescribed and your insurance coverage (if you have it).

The current list price of Zolgensma is over $2 million, making it one of the most expensive drugs in both the U.S.A. and the world. But unlike most drugs, Zolgensma is a one-time treatment. So while the cost per dose is extremely high, the overall treatment cost for Zolgensma is less than other treatments for SMA that are taken for life.

Like most drugs, people prescribed Zolgensma aren’t expected to pay the full list price out of pocket.

To learn more about the cost you may pay for Zolgensma, talk with your child’s doctor or their insurance provider.

Why is Zolgensma so expensive?

Gene therapies like Zolgensma have such a high price tag because they have very expensive research costs. The cost to make them is also extremely high. The manufacturing and development cost required for the production of gene therapies is usually much higher compared with “traditional” drugs.

To learn more about what gene therapies are, check out this resource from the Food and Drug Administration (FDA). You can also talk with your child’s doctor to learn more.

For more information on possible cost-saving options, see “Can I get help paying for Zolgensma?” just below.

If you need help covering the cost of Zolgensma or understanding your insurance, check out these websites:

On these sites, you can find insurance information, details on drug assistance programs, and links to savings cards and other services.

If you have questions about how to pay for your child’s prescription, talk with their doctor or insurance provider.

A program for Zolgensma called OneGene may also be available to you. The OneGene program includes a coordinator who will work with your insurance provider (if you have one) to review your cost for the drug. You may also be eligible for the Zolgensma CopayAssist Program as part of the OneGene program. To learn more about this program, call 855-441-4363. Your doctor may also be able to provide more information.

Zolgensma only comes as a brand-name drug. It’s not currently available in a biosimilar version. Biosimilars are drugs that are similar to a brand-name biologic drug or gene therapy (the parent drug). Zolgensma is a kind of gene therapy, and is considered a biologic medication (also called a biologic).

Biologics are made from living cells. It isn’t possible to make an exact copy of these drugs. A generic, on the other hand, refers to drugs made from chemicals. A generic is an exact copy of the active drug in a brand-name drug.

Biosimilars are considered to be just as effective and safe as their parent drug. And like generics, biosimilars usually cost less than brand-name drugs.

To learn more about what exactly gene therapies are, check out this resource from the Food and Drug Administration (FDA). You can also talk with your doctor to learn more.

Why is there such a cost difference between biologic drugs and biosimilar drugs?

Biologic drugs can be expensive because of the research and testing needed to ensure their safety and effectiveness. The drugmaker of a biologic drug can sell it for up to 12 years. When the biologic drug’s patent expires, other drugmakers can create biosimilar versions. This competition in the market may lead to lower costs for biosimilars. And because biosimilars are very similar to biologic drugs, they don’t need to be studied again. This can also lead to lower costs for biosimilars.

If you still have questions about the cost of Zolgensma treatment, talk with your doctor or pharmacist. They may be able to give you a better idea of what you’ll pay. But if you have health insurance, you’ll need to talk with your insurance provider to learn the actual cost you’d pay for Zolgensma.

Examples of questions you may want to ask your child’s doctor or insurance provider include:

  • What are my child’s options if I can’t afford Zolgensma?
  • How does Zolgensma’s cost compare with other SMA treatments, such as Spinraza?
  • Can you tell me more about options for paying for Zolgensma’s costs over time?

Disclaimer: Healthline has made every effort to make certain that all information is factually correct, comprehensive, and up to date. However, this article should not be used as a substitute for the knowledge and expertise of a licensed healthcare professional. You should always consult your doctor or another healthcare professional before taking any medication. The drug information contained herein is subject to change and is not intended to cover all possible uses, directions, precautions, warnings, drug interactions, allergic reactions, or adverse effects. The absence of warnings or other information for a given drug does not indicate that the drug or drug combination is safe, effective, or appropriate for all patients or all specific uses.