Onasemnogene abeparvovec-xioi (Zolgensma®)

Covered: a single intravenous lifetime dose of Zolgensma for pediatric patients under 2 years with SMA caused by bi‑allelic SMN1 mutations; re‑dosing is not covered and patients with >3 SMN2 copies, baseline anti‑AAV9 antibody titer >1:50, prior Zolgensma, or those >2 years are ineligible. Key requirements: genetic confirmation of bi‑allelic SMN1 mutation (homozygous deletion/mutation or compound heterozygous), age <2 years (premature infants must reach 39 weeks GA), ≤3 SMN2 copies, anti‑AAV9 ≤1:50, baseline LFTs/platelets/troponin‑I, mandatory corticosteroids (prednisolone 1 mg/kg/day starting 1 day before and for 30 days), specialist prescriber/consult, weight‑based dose 1.1×10^14 vg/kg, and discontinuation of Spinraza or Evrysdi if previously treated.

"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 (FDA‑approved indication)."