Onasemnogene abeparvovec-xioi (Zolgensma®)

Covered: a single lifetime IV dose of onasemnogene abeparvovec‑xioi (1.1×10^14 vg/kg) is authorized for pediatric patients <2 years with genetically confirmed bi‑allelic SMN1 mutations and ≤3 SMN2 copies (or exactly 4 only if a quantitative assay distinguishes 4 vs ≥5); excluded are patients ≥2 years, those with anti‑AAV9 titers >1:50, prior Zolgensma, or not meeting SMN criteria. Key requirements: documentation of genetic testing, SMN2 copy number, anti‑AAV9 ≤1:50, baseline labs (LFTs, creatinine, CBC with Hgb/platelets, Troponin‑I), steroid prophylaxis (prednisolone 1 mg/kg/day starting 1 day prior for 30 days), discontinuation of nusinersen/risdiplam if applicable, and prescription/consult by an SMA/neuromuscular specialist.

"Zolgensma is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutati..."