Zolgensma dosage: A detailed guide

This article describes Zolgensma’s dosage and strength and gives details on how it’s given. You’ll also find information on cost savings and coupon options for Zolgensma. If you want to know more about Zolgensma, see this overview article. It covers details about the drug’s uses, side effects, and more.

Zolgensma is a brand-name biologic drug that comes as a suspension for injection into your vein. (A biologic drug is a drug that is sourced from living organisms.) Zolgensma isn’t available in a biosimilar version. This article describes dosages of Zolgensma.

Boxed warning: Risk of liver damage

Zolgensma has a boxed warning for the risk of serious and acute liver damage. A boxed warning is the most serious warning from the Food and Drug Administration (FDA).

See the end of this article for more information about this warning.

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Form and strength of Zolgensma

Zolgensma comes as follows:

• Form: suspension for injection into your vein provided in a kit containing 2 to 14 vials
• Strength: 2 x 10¹³ vector genomes (vg) per milliliter (mL) in a 5.5 mL vial or an 8.3 mL vial

In people with spinal muscular atrophy, the SMN1 gene is not working. Zolgensma is made up of a working SMN1 gene. This gene is placed in a delivery vehicle called a vector. A combination of the gene and vector is called a vector genome.

Dosage of Zolgensma

This article describes the recommended dosage for Zolgensma. The drugmaker provides this dosage.

If your doctor prescribes this medication, you should follow the dosage they prescribe. Your doctor will determine the dosage that meets your child’s needs. Do not change your child’s dosage of Zolgensma without their doctor’s recommendation.

Dosage of Zolgensma in children

Zolgensma is a gene therapy approved to treat spinal muscular atrophy in children younger than ages 2 years.

Zolgensma is made up of a working SMN1 gene. This gene is placed in a delivery vehicle called a vector. A vector containing the SMN1 gene is called a vector genome.

The recommended dosage of Zolgensma is 1.1 x 10¹⁴ vector genomes (vg) per kilogram (kg) of body weight. A kg is approximately 2 pounds. Zolgensma is taken as a single dose. It’s given as an intravenous (IV) infusion (injection into your vein given over time) by a healthcare professional. The IV infusion is given slowly over 60 minutes.

Your child’s doctor will also prescribe a corticosteroid medication to be given for 30 days. The first dose will be given 1 day prior to Zolgensma therapy. After 30 days of corticosteroid therapy, blood tests will be done to check how well the child’s liver is working.

Dosage adjustments for Zolgensma

Zolgensma dosage adjustments are generally not recommended. But your child’s doctor may postpone their dosage of Zolgensma based on several factors. These include:

• the child’s age
• how well the child’s liver works
• other health conditions the child may have, like infections

Missed dose of corticosteroid following Zolgensma infusion

The first dose of corticosteroid medication will be given 1 day prior to Zolgensma therapy. Your child will receive their dose of Zolgensma as a one-time IV infusion. Then, the child must take corticosteroid medication for a total of 30 days.

Talk with your child’s doctor or pharmacist right away if you realize you forgot to give your child a dose of the corticosteroid that must be taken because of Zolgensma. They may advise you to give the child the missed dose. Or they may advise you to skip it and give the child their next dose as scheduled.

For tips on how to plan your doses and avoid missing a dose, read this article. You could also try:

• downloading a reminder app on your phone
• setting an alarm
• putting a note where you’ll see it, such as on your calendar, bathroom mirror, or bedside table

Frequently asked questions

Below are answers to some commonly asked questions about Zolgensma and its dosage.

How does the dosage of Zolgensma compare with the dosage of Spinraza?

Doctors use Spinraza to treat spinal muscular atrophy in children and adults. Zolgensma and Spinraza are both given by healthcare professionals. But they are given via different routes, and they have different schedules. Zolgensma is a one-time injection given into the child’s vein over 60 minutes. Your child will need to take a corticosteroid medication for 30 days starting 1 day prior to their Zolgensma infusion.

Doctors give Spinraza intrathecally (an injection into your spinal fluid). Doses are 12 milligrams (mg) each, with the dose given over a few minutes. When Spinraza therapy starts, three doses are given 14 days apart. Then, 30 days after the third dose, a fourth dose is given. You will then transition to maintenance dosing, which is once every 4 months.

Keep in mind that Zolgensma and Spinraza work differently. So, one of these medications may be more appropriate for your child’s condition.

Speak with your child’s doctor to learn more about which medication is best for your child and what their dosage will be.

Is Zolgensma used long term?

No, doctors don’t prescribe Zolgensma as a long-term treatment. It’s a single dose given as a one-time IV infusion.

If you have questions about how long Zolgensma is used, talk with your child’s doctor.

How Zolgensma is administered

Your child will be given Zolgensma once as an infusion into their vein over 60 minutes. Your child will also be given a corticosteroid to take starting 1 day prior to the Zolgensma infusion. This corticosteroid medication should be taken for 30 days.

Your child’s doctor may advise that they take this corticosteroid medication around the same time each day. This helps keep a consistent amount of the drug in your child’s body. And that can help the drug work more effectively.

Also, be sure to talk with your doctor or pharmacist if your child is having difficulty taking the corticosteroid medication. They can offer recommendations about taking it.

Zolgensma coupon

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What to ask your doctor

This article describes the usual recommended dosage for Zolgensma. If your doctor prescribes this medication, they’ll determine the dosage that’s best for your child.

Your child will be given Zolgensma exactly as your doctor prescribes it. Let your child’s doctor know if you have concerns or questions about the treatment plan.

Here’s a list of some possible questions you may want to ask your child’s doctor:

• What lab work should be performed before and after my child’s Zolgensma infusion?
• Will my child’s vaccination schedule change to allow them to receive Zolgensma?
• How will I know whether Zolgensma worked for my child?

Boxed warning for Zolgensma

Zolgensma has a boxed warning for the risk of liver damage. A boxed warning is the most serious warning from the FDA.

Serious and acute liver damage: Zolgensma can cause serious and acute liver damage that can be life threatening. Your child may be at increased risk for liver damage if they already have decreased liver function. Prior to receiving Zolgensma, your child’s doctor will check how well their liver works. Your child will also take a corticosteroid for 30 days, which will help prevent liver damage. Your doctor will continue to monitor how well your child’s liver is working for at least 3 months after Zolgensma infusion. Seek medical attention if your child experiences vomiting or overall worsening of their health.

Talk with your doctor to learn more about this warning.

Disclaimer: Optum Perks 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.