Neurology – Skysona®

Skysona is covered as a one-time (per lifetime) intravenous gene therapy for biologic males ≥4 and <18 years with early, active cerebral adrenoleukodystrophy and is excluded for patients with full ABCD1 deletions, prior HSCT or gene therapy, prior/current hematologic malignancy or familial cancer syndrome, active infection, advanced disease, or otherwise not meeting criteria; a boxed warning notes risk of post‑treatment hematologic malignancy. Approval requires documentation of neurologic function score ≤1, gadolinium‑enhancing MRI lesions, Loes score 0.5–9, pathogenic ABCD1 variant and elevated VLCFA, either no HLA‑matched donor or donor unwilling, negative pre‑collection infectious screens (HBV, HCV, HTLV‑1/2, HIV), specified liver/renal/hematologic lab thresholds within 30 days, planned mobilization/apheresis/myeloablative conditioning and lymphodepletion using G‑CSF, busulfan and cyclophosphamide or fludarabine, specialist prescribing, verification of no prior Skysona, and dosing ≥5.0×10^6 CD34+ cells/kg.