Spinal Muscular Atrophy - Zolgensma®

Zolgensma is covered as a one‑time (per lifetime) IV dose (1.1×10^14 vg/kg) for patients <2 years with genetically confirmed bi‑allelic SMN1 pathogenic variants who meet SMN2 copy criteria (≤3 copies or 4 copies confirmed by a quantitative assay), baseline anti‑AAV9 ≤1:50, recent weight, specialist prescribing with confirmation that Spinraza/Evrysdi will be discontinued if applicable, and required labs within 30 days (ALT/AST/total bilirubin/PT ≤2×ULN, creatinine <1.0 mg/dL, WBC ≤20,000, Hgb 8–18 g/dL) plus verification of no prior gene therapy. Coverage excludes patients ≥2 years, those with complete limb paralysis, permanent ventilator dependence, prior gene therapy, in utero administration, or premature neonates before 39 weeks post‑menstrual age; approvals require claims/medical director verification and must be administered within a 30‑day authorization window.

"Treatment of spinal muscular atrophy with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene in patients who are < 2 years of age."