A Treatment for Spinal Muscular Atrophy (SMA)
ZOLGENSMA® is the brand name for onasemnogene abeparvovec-xioi, a gene therapy approved by the Food and Drug Administration (FDA) May 24th, 2019. It is approved for children less than 2 years old with spinal muscular atrophy (SMA).
Frequently Asked Questions About ZOLGENSMA®
Is my child eligible for treatment with ZOLGENSMA®?
Your child must have the survival motor neuron 1 (SMN1) genetic mutation that causes SMA. A genetic confirmation is necessary to confirm your child’s eligibility.
An AAV9 antibody test may be required by some insurance providers. An AAV9 antibody test measures the amount of anti-AAV9 antibodies in your child’s blood. If your child’s immune system has built up a certain level of anti-AAV9 antibodies, he or she may not qualify for ZOLGENSMA® right away.
How does ZOLGENSMA® work?
Patients with SMA are born without the SMN1gene. Instead, they have two copies of survival motor neuron 2 (SMN2). Unfortunately, there is a problem with a piece of protein in SMN2.
As a gene therapy, ZOLGENSMA® is designed to target the genetic root cause of SMA by replacing the function of the missing or nonworking SMN1gene with a new, working copy of a human SMN gene. Once infused, the vector delivers the healthy SMN1gene to cells throughout the body. When the healthy SMN1 gene reaches a child’s cells, it begins producing normally-functioning SMN protein, thereby improving motor neuron function and survival.
How is ZOLGENSMA® given to patients?
ZOLGENSMA® is given as a one-time dose by an intravenous (IV) infusion that takes about 60 minutes. A course of an oral corticosteroid should be started the day before the infusion and your child will continue with it as prescribed.
What is the benefit of ZOLGENSMA®?
Clinical trials have shown clear efficacy in young children with SMA type 1, resulting in a decreased need for respiratory support as well as improvement in motor skills. Trials have also shown that the earlier children receive gene therapy, the better the results.
Is ZOLGENSMA® covered by insurance?
Your doctor will need to submit a ZOLGENSMA® Prescription Form and a Patient Consent Form to the OneGene Program to start the process to assess insurance coverage and authorization requirements. The OneGene Program provides support along the way. It is a dedicated resource brought to you by AveXis and is specifically designed to help you and your family.