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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7 Key Takeaways

  • 1

    Itvisma is a gene replacement therapy approved for SMA.

  • 2

    Approved for patients 2 years and older with SMN1 mutation.

  • 3

    Administered as a one-time intrathecal injection.

  • 4

    Significant motor function improvement noted in clinical trials.

  • 5

    The therapy offers support services for patient initiation.

  • 6

    Common adverse events include respiratory infections and fever.

  • 7

    Available in December, targeting unmet needs in SMA.

Original Source(s)

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.

  • by Kathryn Wighton

  • November 25, 2025

  • 2 min

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