Spinal Muscular Atrophy - Zolgensma®

Covers a single lifetime IV dose of Zolgensma for pediatric patients <2 years with genetically confirmed bi‑allelic SMN1 pathogenic variants who meet SMN2 copy‑number criteria, and excludes patients with complete limb paralysis, permanent ventilator dependence, in‑utero administration, prior Zolgensma receipt, repeat dosing, or those who fail specified lab/antibody thresholds. Approval requires documentation of SMN2 copy number (≤3 or 4 confirmed by a quantitative assay), baseline anti‑AAV9 ≤1:50, liver/renal/CBC within specified limits, current weight for dosing (1.1×10^14 vg/kg), specialist prescriber, a corticosteroid regimen (prednisolone 1 mg/kg/day starting 1 day prior for 30 days), discontinuation of other SMA disease‑modifying therapies, and claims/physician confirmation of no prior Zolgensma.

"FDA‑approved indication: 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."