The FDA has approved Itvisma (intrathecal onasemnogene abeparvovec) as a gene-replacement therapy for spinal muscular atrophy (SMA) in patients aged two and older, expanding access beyond infant indications. Delivered via a single intrathecal injection to the central nervous system, the therapy targets SMN gene deficiency that leads to progressive motor neuron loss. Novartis positioned Itvisma as a spectrum-wide option, citing clinical data supporting functional benefits across age and severity ranges. FDA leadership framed the approval as a milestone for neurological gene medicine, while emphasizing continued safety monitoring given the complexity of CNS delivery. The news follows Novartis’ recent manufacturing investments and acquisitions, signaling a broader strategic push in genetic therapies and neurodegeneration.
Source: NJBIZ (Dec 2, 2025)
Link: https://njbiz.com/novartis-itvisma-fda-approval-sma-gene-therapy/
FDA Approves Novartis’ Itvisma, Expanding Gene Therapy for SMA
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