FDA approves muscle-targeted SMA therapy - Takeaways - MDSpire
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FDA approves muscle-targeted SMA therapy

The therapy inhibits myostatin signaling and is used with survival motor neuron 2-targeted treatment.

  • By

  • Kathryn Wighton

  • September 14, 2026

  • 2 min

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    The FDA approved apitegromab-mstn (Isembyld) for spinal muscular atrophy in patients aged 2 years and older.

  • 2

    Apitegromab is a monoclonal antibody designed to inhibit myostatin signaling in spinal muscular atrophy.

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    The approval was based on the SAPPHIRE trial, which included 188 patients aged 2 to 21 years with 5q SMA.

  • 4

    Patients receiving apitegromab 10 mg/kg showed a 2.2-point improvement on the Hammersmith scale compared to placebo.

  • 5

    Common adverse reactions included upper respiratory infections, vomiting, and fractures in 9% of patients on apitegromab.

Original Source(s)

FDA approves muscle-targeted SMA therapy

  • by Kathryn Wighton

  • September 14, 2026

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