Zolgensma® (onasemnogene abeparvovec-xioi)

Covered: a single one-time IV dose of Zolgensma for pediatric patients <2 years with genetically confirmed bi-allelic SMN1 pathogenic variants (excludes patients ≥2 years, those without bi-allelic SMN1, prior Zolgensma recipients, baseline anti‑AAV9 >1:50, or those who fail required lab/assay criteria). Key requirements: documentation of genetic testing and SMN2 copy number (≤3 copies, or 4 copies confirmed by an assay that distinguishes 4 vs ≥5), baseline anti‑AAV9 ≤1:50, labs within 30 days (creatinine <1.0 mg/dL; ALT/AST/total bilirubin/PT ≤2×ULN; WBC ≤20,000; Hgb 8–18 g/dL), discontinuation of Spinraza/Evrysdi if applicable, systemic corticosteroids (prednisolone 1 mg/kg/day starting 1 day before and for 30 days), prescription/consultation by an SMA/neuromuscular specialist, dosing 1.1×10^14 vg/kg, one dose per lifetime, and premature neonates must have postmenstrual age ≥39 weeks.

"Treatment of spinal muscular atrophy (SMA) — approval granted only when the individual meets all required coverage guideline criteria (see Limitations and Documentation Requirements)."