Zolgensma

Generic name: Onasemnogene Abeparvovec-xioi
Dosage form: intravenous (infusion) injection
Drug class: Miscellaneous uncategorized agents

Usage of Zolgensma

Zolgensma is a gene therapy used for spinal muscular atrophy (SMA). SMA is an inherited disorder that causes skeletal muscle weakness and wasting, which worsens with age.

SMA is caused when a gene, usually SMA1, is missing or doesn't work properly. Zolgensma provides a new functional or working copy of a human SMN gene.

Zolgensma is a one-time treatment given via an intravenous (IV) infusion. It was approved by the US Food and Drug Administration (FDA) in 2019.

Zolgensma side effects

The most common side effects that occurred in patients treated with Zolgensma were elevated liver enzymes and vomiting.

These are not all of the possible side effects with Zolgensma. Call your child's doctor for medical advice about side effects that bother you or do not go away.

You are encouraged to report suspected side effects by contacting the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch, or Novartis Gene Therapies, Inc. at 833-828-3947.

Before taking Zolgensma

Before receiving Zolgensma, talk with your child's doctor about:

Vaccinations to decide if adjustments to the vaccination schedule are needed to accommodate treatment with a corticosteroid.

Protection against respiratory syncytial virus (RSV), which is recommended.

Relate drugs

How to use Zolgensma

The recommended dosage of Zolgensma is 1.1 × 1014 vector genomes (vg) per kg of body weight.

Warnings

Zolgensma can increase liver enzyme levels and cause acute serious liver injury or acute liver failure.

Your child will receive an oral corticosteroid before and after infusion with Zolgensma and will undergo regular blood tests to monitor liver function.

Contact your child's doctor immediately if the patient’s skin and/or whites of the eyes appear yellowish, if they miss a dose of corticosteroid or vomits it up, or if the patient experiences a decrease in alertness.

What other drugs will affect Zolgensma

Adjustments may need to be made to your child's vaccination schedule to accommodate treatment with a corticosteroid. Talk with your child's doctor.

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