Is Zolgensma a cure for SMA?

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:

• Evrysdi (risdiplam)
• Spinraza (nusinersen)
• Zolgensma (onasemnogene abeparvovec)