Start treating your patients with ZOLGENSMA
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. ZOLGENSMA is given as a one-time infusion into a vein. ZOLGENSMA was not evaluated in patients with advanced SMA.1
To prescribe ZOLGENSMA to a patient, complete a Novartis Patient Support Start Form and have the parent or legal guardian complete the Novartis Patient Support Patient Authorization and Additional Consents Form. A signed Patient Authorization and Additional Consents Form and completed Start Form are needed for patients and families to receive support through Novartis Patient SupportTM.
Novartis Patient Support Start Form and Patient Authorization and Additional Consents Form
Completed forms must be submitted for enrollment SUBMIT ONLINE
Pre-treatment requirements
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 or treating physician.1
Genetic confirmation1
A genetic diagnosis of SMA
Confirmation of the bi-allelic mutation in SMN1 gene
SMN2 copy number analysis is recommended
AAV9 antibodies1
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 elevated
Baseline tests1
Baseline evaluations of liver function (clinical exam, AST, ALT, total bilirubin, albumin, prothrombin time, PTT, and INR), creatinine, and complete blood count (including hemoglobin and platelet count)
Liver function and platelet count will need to be monitored following infusion
Consider cardiac evaluation after infusion and consult a cardiologist as needed
Administer ZOLGENSMA to patients who are clinically stable in their overall baseline health status (eg, hydration and nutritional status, absence of infection) prior to infusion. Postpone ZOLGENSMA in patients with infections until the infection has resolved and the patient is clinically stable. Clinical signs or symptoms of infection should not be evident at the time of ZOLGENSMA infusion.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 gestational age is reached.1
The safety and effectiveness of repeat administration or the use in patients with advanced SMA (eg, complete paralysis of limbs, permanent ventilator dependence) has not been evaluated with ZOLGENSMA.1
Importance of early treatment
Learn from accomplished neuromuscular specialists about the urgency to treat patients with SMA as soon as possible and how to anticipate common barriers to treatment.