FDA OKs First SMA Gene Replacement Therapy - Takeaway - MDSpire
FDA & Government News

FDA OKs First SMA Gene Replacement Therapy

The FDA approved Itvisma, the first gene replacement therapy for children 2 years and older, teens, and adults with spinal muscular atrophy, a one-time treatment that replaces the SMN1 gene and demonstrated improved motor function in Phase 3 studies.

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    Itvisma is a gene replacement therapy approved for SMA.

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    Approved for patients 2 years and older with SMN1 mutation.

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    Administered as a one-time intrathecal injection.

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    Significant motor function improvement noted in clinical trials.

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    The therapy offers support services for patient initiation.

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    Common adverse events include respiratory infections and fever.

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    Available in December, targeting unmet needs in SMA.

Original Source(s)

FDA OKs First SMA Gene Replacement Therapy

  • by Kathryn Wighton

  • November 25, 2025

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