Neurology Skysona®

Covers a one‑time (per lifetime) single IV dose of Skysona for male patients 4 to <18 years with early, active cerebral adrenoleukodystrophy and excludes those with full ABCD1 deletion, prior HSCT or prior gene therapy, active infection, or prior/current hematologic malignancy/familial cancer syndromes; Skysona carries a boxed warning for risk of hematologic malignancy. Approval requires strict documentation of NFS ≤1, MRI gadolinium enhancement, Loes score 0.5–9, pathogenic ABCD1 variant, elevated VLCFA, acceptable hepatic/renal/hematologic labs and negative HBV/HCV/HTLV/HIV screens within 30 days, verification of no prior Skysona and lack of a willing HLA‑matched donor, a treatment plan using G‑CSF mobilization, apheresis, busulfan conditioning and cyclophosphamide or fludarabine lymphodepletion, prescribing by an appropriate specialist, and a product dose ≥5.0×10^6 CD34+ cells/kg (authorization up to 6 months for preparation).

"Cerebral adrenoleukodystrophy (early, active) — approval of a one-time (per lifetime) single intravenous dose of Skysona (elivaldogene autotemcel) is recommended when ALL of the following are met:"