FDA Approves Itvisma: New Gene Therapy for Spinal Muscular Atrophy (SMA) in Patients 2+ Years
The U.S. Food and Drug Administration has approved Itvisma (onasemnogene abeparvovec-brve), an adeno-associated virus (AAV) vector–based gene therapy for adults and pediatric patients 2 years of age and older with spinal muscular atrophy (SMA) caused by a confirmed mutation in the SMN1 gene.
“Today’s approval shows the power of gene therapies and offers treatment to patients across the SMA disease spectrum,”
— Vinay Prasad, M.D., M.P.H., FDA Chief Medical and Scientific Officer
About Spinal Muscular Atrophy (SMA)
SMA is a genetic neurodegenerative disorder caused by mutations in the SMN1 gene. The disease leads to progressive motor neuron loss, severe muscle weakness, paralysis, and in the most critical cases, early death. SMA affects 4–10 per 10,000 live births and was historically a leading cause of infant genetic mortality in the U.S.
How Itvisma Works
Itvisma is a concentrated formulation of the same active ingredient found in Zolgensma, but delivered differently:
- Route of administration: A single intrathecal injection (directly into the cerebrospinal fluid)
- Dose: Independent of patient weight
- Target: Motor neurons in the spinal cord
- Mechanism: Restores SMN protein production to halt further disease progression
Direct CNS delivery allows effective treatment with a lower vector dose and rapid onset of action.
Clinical Evidence Supporting Approval
FDA approval is based on:
- A controlled Phase 3 study in pediatric patients aged 2+
- Mechanistic evidence demonstrating effective gene expression
- Confirmatory safety and efficacy data from Zolgensma
The FDA also reviewed safety data from Zolgensma and retained hepatotoxicity warnings in Itvisma’s label, with modifications based on clinical findings.
The applicant provided justification to extend the indication to adult SMA patients, though additional precautions apply for individuals with chronic medical conditions.
A New Option for Older SMA Patients
Previously, Zolgensma was limited to patients under 2 years of age.
Itvisma expands gene therapy access to:
- Children over 2
- Adolescents
- Adults with SMA
This marks a significant milestone for patients who previously had limited gene therapy options.
FDA Designations
To accelerate development, Itvisma received:
- Fast Track
- Breakthrough Therapy
- Priority Review
- Orphan Drug Designation
Manufacturing
Itvisma is manufactured by Novartis Gene Therapies, Inc.
For more details, visit the official FDA announcement:
https://www.fda.gov/news-events/press-announcements/fda-approves-gene-therapy-treatment-spinal-muscular-atrophy
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