Zolgensma® (onasemnogene abeparvovec-xioi)

Zolgensma is covered as a single lifetime IV dose (1.1×10^14 vg/kg) for pediatric patients <2 years with genetically confirmed SMA (bi‑allelic SMN1 pathogenic variants) who meet SMN2 copy‑number criteria (≤3 copies, or 4 copies only if quantified by an assay that distinguishes 4 vs ≥5) and have not previously received Zolgensma. Approval requires documented baseline anti‑AAV9 titer ≤1:50, labs within 30 days (creatinine <1.0 mg/dL; ALT/AST/total bilirubin/PT ≤2×ULN; CBC with WBC ≤20,000 and Hgb 8–18 g/dL), SMA specialist prescribing/consultation, discontinuation plans for nusinersen/risdiplam if applicable, and a corticosteroid plan (prednisolone 1 mg/kg/day starting 1 day prior and continued for 30 days).

"Zolgensma is indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene."