Zolgensma® (onasemnogene abeparvovec-xioi)

Covered: a single lifetime IV dose of Zolgensma (1.1×10^14 vg/kg) is covered for pediatric patients <2 years with genetically confirmed SMA due to bi‑allelic SMN1 mutations; coverage is excluded for prior Zolgensma recipients, patients ≥2 years, those with baseline anti‑AAV9 titers >1:50, or those who cannot meet eligibility criteria. Key requirements: documentation of SMN2 copy number (≤3, or 4 only if a quantitative assay distinguishes 4 vs ≥5), anti‑AAV9 ≤1:50, renal/liver/CBC labs within 30 days meeting specified thresholds (creatinine <1.0 mg/dL; ALT/AST/total bilirubin/PT ≤2×ULN; WBC ≤20,000; Hgb 8–18 g/dL), plan to stop Spinraza/Evrysdi if applicable, systemic corticosteroids (prednisolone 1 mg/kg/day starting 1 day prior for 30 days), and prescription by or consultation with an SMA/neuromuscular specialist.

"Baseline anti-AAV9 antibody titers must be ≤ 1:50; higher titers are excluded."