Before treatment

Start treating your patients with ZOLGENSMA:

Identifying appropriate patients

ZOLGENSMA is approved for pediatric patients with SMA less than 2 years of age, who are genetically confirmed for bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. The safety and effectiveness of repeat administration or the use in patients with advanced SMA (e.g., complete paralysis of limbs, permanent ventilator dependance) has not been evaluated with ZOLGENSMA.1

Use of ZOLGENSMA in premature neonates before reaching full term gestational age is not recommended because concomitant treatment with corticosteroids may adversely affect neurological development. Delay ZOLGENSMA infusion until full-term gestation age is reached.1

Once you and your patient’s caregiver have decided on ZOLGENSMA, the following tests need to be performed prior to infusion. These tests can be ordered by the referring physician or a physician at a ZOLGENSMA-ready institution.

AveXis is offering the AveXis Laboratory Program to assist with genetic and anti-AAV9 antibody testing
For more information, please contact your AveXis Key Account Manager or call the OneGene Program™ at
1-855-441-GENE (4363)

Genetic Confirmation1

  • Confirmation of the bi-allelic mutation in SMN1 gene
  • SMN2 copy number analysis2

AAV9 Antibody Test1

  • Testing for the presence of anti-AAV9 antibodies
  • The safety and efficacy of ZOLGENSMA in patients with anti-AAV9 antibody titers above 1:50 have not been evaluated
  • Retesting may be performed if anti-AAV9 antibody titers are reported as positive or elevated1

Baseline Tests1

  • Baseline evaluations of liver function,a platelet count, and troponin-I
  • These values will need to be monitored following infusion

Report test results to the OneGene Program
at 1-855-441-GENE (4363)

Referring patients for treatment

Early treatment is essential for infants with SMA.3 If your institution is not ready to infuse ZOLGENSMA, refer your eligible patients for treatment. Work with your patients’ families to help identify the ZOLGENSMA-ready institution that is right for them.

To locate a treatment center that can administer ZOLGENSMA:
email treatments@curesma.org

OR

contact the MDA Resource Center at 1-833-ASK-MDA1 or resourcecenter@mdausa.org

The ZOLGENSMA Prescription and Patient Consent Forms

To begin prescribing ZOLGENSMA for your patients, complete a ZOLGENSMA Prescription Form and accompanying Patient Consent Form. A signed Patient Consent Form is needed for patients and families to receive support through the OneGene Program.

Please fax the signed ZOLGENSMA Prescription Form and Patient Consent Form as soon as they have been completed to 1-855-951-GENE (4363)