FDA OKs First SMA Gene Replacement Therapy - Summary - 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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Onasemnogene abeparvovec-brve, marketed as Itvisma, has been approved by the FDA for individuals aged 2 and older with spinal muscular atrophy (SMA) due to an SMN1 mutation. This innovative gene replacement therapy, administered via a one-time intrathecal injection, aims to enhance motor function by delivering a functional SMN1 gene. Clinical trials including the Phase 3 STEER and Phase 3b STRENGTH studies demonstrated significant improvements in motor function over 52 weeks. With an estimated 9,000 affected patients in the US, this therapy addresses unmet needs especially in older children and adults. Itvisma is set to be available in December, accompanied by support services from Novartis.

Original Source(s)

FDA OKs First SMA Gene Replacement Therapy

  • by Kathryn Wighton

  • November 25, 2025

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