Neurology – Skysona®

Coverage: one lifetime IV dose of Skysona is covered for male patients 4 to <18 years with early, active cerebral adrenoleukodystrophy (NFS=1, gadolinium enhancement on MRI, Loes score 0.5–9); excluded for full ABCD1 gene deletion, prior HSCT or prior gene therapy, presence of an HLA‑matched family donor, active infection, prior/current hematologic malignancy or familial cancer syndrome, or other unmet criteria. Key requirements: documented ABCD1 mutation and elevated VLCFA, specified hepatic/renal/hematologic lab thresholds and negative infectious disease screens, documented plan for cell mobilization/apheresis, myeloablative conditioning with busulfan, lymphodepletion with cyclophosphamide or fludarabine and G‑CSF mobilization, VOD prophylaxis, specialist prescriber, contraception from mobilization through 6 months, minimum dose 5.0 x 10^6 CD34+ cells/kg, prior authorization recommended, approval limited to one dose per lifetime with a 6‑month authorization window.