Spinal Muscular Atrophy (SMA) Treatments
Find Spinal Muscular Atrophy (SMA) Treatments
Medications for Spinal Muscular Atrophy (SMA)
These are drugs that have been approved by the US Food and Drug Administration (FDA), meaning they have been determined to be safe and effective for use in Spinal Muscular Atrophy (SMA).
Found 2 Approved Drugs for Spinal Muscular Atrophy (SMA)
Zolgensma
Generic Name
Onasemnogene Abeparvovec-Xioi
Zolgensma
Generic Name
Onasemnogene Abeparvovec-Xioi
Form: Kit
FDA approval date: May 24, 2019
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.
Itvisma
Generic Name
Onasemnogene Abeparvovec-brve
Itvisma
Generic Name
Onasemnogene Abeparvovec-brve
Form: Injection
Method of administration: Intrathecal
FDA approval date: November 24, 2025
ITVISMA is indicated for the treatment of spinal muscular atrophy (SMA) in adult and pediatric patients 2 years of age and older with confirmed mutation in survival motor neuron 1 (SMN1) gene. ITVISMA is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of spinal muscular atrophy (SMA) in adult and pediatric patients 2 years of age and older with confirmed mutation in SMN1 gene. ( 1 )
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