FDA OKs First SMA Gene Replacement Therapy
November 25, 2025
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2 min
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.
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.
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