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Is Zolgensma a cure for SMA?

Medically reviewed by Leigh Ann Anderson, PharmD. Last updated on Dec 30, 2024.

Official answer

by Drugs.com

No, Zolgensma (onasemnogene abeparvovec-xioi) is not a cure for SMA (spinal muscular atrophy). In addition, it cannot reverse any damage to motor neurons already caused by SMA before treatment.

Since damage is not reversible, early treatment with Zolgensma is very important. In studies in children with SMA, Zolgensma has been shown to help stop the progression of the disease. Early treatment can help to preserve motor neuron cells and muscle function needed to survive.

Zolgensma is given as a one-time intravenous (IV) infusion that takes about one hour and is given at a treatment center.

Zolgensma has been shown to:

  • improve muscle strength
  • increase survival without the need for permanent breathing support
  • maintain the ability to thrive (which includes swallowing function, freedom from non-oral feeding support like a gastrostomy tube, and maintaining weight gain that is consistent for the child’s age)

Zolgensma was approved by the FDA in May 2019 to treat children less than 2 years of age with SMA mutations in the survival motor neuron 1 (SMN1) gene. Zolgensma has not been studied in children with advanced SMA (for example, with complete paralysis of limbs or permanent ventilator dependence).

Spinal muscular atrophy (SMA) is a rare genetic motor neuron disease that leads to problems with breathing, holding up the head, and swallowing. The majority of children with SMA do not survive past early childhood due to respiratory failure (inability to breathe). In its most severe form, SMA can lead to permanent ventilation or death by age two.

What is the mechanism of action of Zolgensma?

Zolgensma (onasemnogene abeparvovec-xioi) is a recombinant adeno-associated virus vector-based (AAV9-based) gene therapy. It works by using the AAV9 vector to help carry the SMN1 gene into the body to replace the function of the missing or nonworking SMN1 gene to the motor neuron cells.

SMA is caused by a mutation in the survival motor neuron 1 (SMN1) gene, which results in low or missing (survival motor neuron (SMN) protein expression.

This is not all the information you need to know about Zolgensma (onasemnogene abeparvovec-xioi) for safe and effective use and does not take the place of talking to your doctor about your treatment. Review the full Zolgensma information here, and discuss this information and any questions you have with your doctor or other health care provider.

Which medicines treat Spinal Muscular Atrophy (SMA)?

The drug treatments available to treat SMA include:

References

Read next

Why is Zolgensma so expensive?

Zolgensma costs $2.1 million for the one-time-only treatment. The reason Zolgensma is so expensive is because that is the price Novartis has decided it is worth because it “dramatically transforms the lives of families affected by this devastating disease” and the claimed cost of bringing new drugs to market. But this price is not without controversy. Continue reading

Zolgensma vs Spinraza: What are the key differences?

Zolgensma (onasemnogene abeparvovec-xioi) and Spinraza (nusinersen sodium) are both treatments for people with spinal muscular atrophy (SMA). There are a number of key differences between the two treatments, including:

  1. Which patients with SMA they are approved for use in
  2. How often they are administered
  3. The type of treatments they are
  4. How they work
  5. How well they work
Continue reading

How is Zolgensma administered?

Zolgensma is given as a one-time intravenous (IV) infusion into a vein at a treatment center. The infusion takes approximately one hour. Your healthcare provider can direct you to the closest treatment center. Continue reading

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