Zolgensma
ONASEMNOGENE ABEPARVOVEC-XIOI
Manufacturer: Novartis Gene Therapies, Inc.
Black Box Warning (Boxed Warning)
A Boxed Warning is the FDA's strongest safety warning — it indicates serious or life-threatening risks identified during clinical trials or post-market surveillance. It does not mean the medication is unsafe for all patients; your prescriber weighs these risks against the benefits for your specific situation. Source: FDA-approved prescribing information.
FDA-Approved Indications (2)
1 INDICATIONS AND USAGE ZOLGENSMA is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (…
Indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.
Indications & Usage
1 INDICATIONS AND USAGE ZOLGENSMA is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Limitations of Use The safety and effectiveness of repeat administration of ZOLGENSMA have not been evaluated [see Adverse Reactions ( 6.2 )] . The use of ZOLGENSMA in patients with advanced SMA (e.g., complete paralysis of limbs, permanent ventilator-dependence) has not been evaluated [see Clinical Studies ( 14 )] . ZOLGENSMA is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 ( SMN1) gene. ( 1 ) Limitations of Use The safety and effectiveness of repeat administration of ZOLGENSMA have not been evaluated. ( 1 , 6.2 ) The use of ZOLGENSMA in patients with advanced SMA (e.g., complete paralysis of limbs, permanent ventilator dependence) has not been evaluated. ( 1 , 14 )
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