FDA Updates SMA Treatment Dosing - Takeaways - MDSpire

FDA Updates SMA Treatment Dosing

A higher-dose nusinersen regimen for spinal muscular atrophy demonstrated statistically significant motor function improvement in treatment-naïve infants in the DEVOTE trial with a safety profile consistent with prior dosing.

  • By

  • Kathryn Wighton

  • March 30, 2026

  • 2 min

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  • 1

    The FDA approved a higher-dose regimen of nusinersen (SPINRAZA) for spinal muscular atrophy in pediatric and adult patients.

  • 2

    The new dosing schedule includes two 50 mg loading doses followed by maintenance doses of 28 mg every 4 months.

  • 3

    Approval was based on the phase 2/3 DEVOTE study, which showed significant motor function improvement in treatment-naïve infants.

  • 4

    The safety profile of the high-dose regimen aligns with the lower-dose regimen, with common adverse events including pneumonia.

  • 5

    Nusinersen is delivered via intrathecal injection and is expected to be available in the US soon, following approvals in other regions.

Original Source(s)

FDA Updates SMA Treatment Dosing

  • by Kathryn Wighton

  • March 30, 2026

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