Start treating your patients with ZOLGENSMA:
If your institution is ready to infuse
Complete the required laboratory tests1:
- Confirm a genetic diagnosis of SMA
- Determine the patient’s anti-AAV9 antibody titers
- Establish baseline values for liver function,a creatinine, complete blood count (including hemoglobin and platelet count), and troponin-I
Learn more about required laboratory testing prior to ZOLGENSMA infusion.
aLiver function assessment includes a clinical exam and laboratory testing of hepatic aminotransferases (aspartate aminotransferase [AST] and alanine aminotransferase [ALT]), total bilirubin, and prothrombin time.
Fax a completed ZOLGENSMA Prescription Form and accompanying Patient Consent Form to the OneGene Program™ at 1-855-951-GENE (4363).
You may send the forms while the required test results are pending. Once complete, report the results to the OneGene Program.
If you would like to refer your patient for treatment
Complete the required laboratory tests1:
- Confirm a genetic diagnosis of SMA
- Determine the patient’s baseline anti-AAV9 antibody titers
Report laboratory test results to the OneGene Program
at 1-855-441-GENE (4363).
Refer your patients for treatment:
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.
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.
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.
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 creatinine, complete blood count (including hemoglobin and platelet count), and troponin-I
- Liver function, platelet countb, and troponin-I will need to be monitored following infusion
aLiver function assessment includes a clinical exam and laboratory testing of hepatic aminotransferases (aspartate aminotransferase [AST] and alanine aminotransferase [ALT]), total bilirubin, and prothrombin time.
bMonitor platelet counts as well as signs and symptoms of TMA, such as hypertension, increased bruising, seizures, or decreased urine output.
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
The safety and effectiveness of repeat administration or the use in patients with advanced SMA (e.g., complete paralysis of limbs, permanent ventilator dependence) has not been evaluated with ZOLGENSMA.1
Novartis Gene Therapies is offering the Novartis Gene Therapies Laboratory Program to assist with genetic and anti-AAV9 antibody testing.
For more information, please contact your Novartis Gene Therapies Account Manager or call 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 prescribe ZOLGENSMA to a patient, 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).