Spinal Muscular Atrophy - Zolgensma®

Approved: one-time, lifetime IV Zolgensma for patients <2 years with genetically confirmed bi-allelic SMN1 pathogenic variants who meet strict criteria — SMN2 copy ≤3 (or a qualifying quantitative 4‑copy result), baseline anti‑AAV9 ≤1:50, ALT/AST/total bilirubin/PT ≤2×ULN, creatinine <1.0 mg/dL, WBC ≤20,000, hemoglobin 8–18 g/dL, documented weight within 14 days, and receipt of corticosteroids (prednisolone 1 mg/kg/day) starting 1 day before infusion for 30 days, with prior authorization and specialist prescribing/consultation and required documentation. Excluded/limited: prior Zolgensma, repeat dosing, in‑utero administration, complete limb paralysis, permanent ventilator dependence, failure to meet age/lab/antibody criteria, or ongoing nusinersen/risdiplam without confirmed discontinuation.

"Spinal Muscular Atrophy - Treatment: Approve for a one-time per lifetime dose for patients who meet the specified criteria (A through N)."