Spinal Muscular Atrophy (SMA) Approved Drugs
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)
Spinraza
Generic Name
Nusinersen
Spinraza
Generic Name
Nusinersen
Form: Injection
Method of administration: Intrathecal
FDA approval date: December 23, 2016
Classification: Antisense Oligonucleotide
SPINRAZA is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. SPINRAZA is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients ( 1 )
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.
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