Neurology - Lenmeldy™

Lenmeldy is covered as a one-time (per‑lifetime) IV infusion for children with presymptomatic late‑infantile (PSLI), presymptomatic early‑juvenile (PSEJ), or early‑symptomatic early‑juvenile (ESEJ) MLD and is excluded for late‑juvenile or adult MLD, GMFC‑MLD > Level 1, prior allogeneic HSCT within 6 months or residual donor cells, prior gene therapy, prior Lenmeldy, or other non‑specified circumstances. Approval requires prior authorization with documented ARSA genotype and low ARSA activity, elevated 24‑hour urine sulfatide, negative infectious screens (HIV/HBV/HCV/HTLV/CMV/mycoplasma), recent weight, specialty prescriber (hematologist/neurologist/medical geneticist/stem‑cell transplant specialist), attested plan for mobilization/apheresis with G‑CSF ± mobilizer and myeloablative conditioning with busulfan, discontinuation of prophylactic antiretrovirals ≥1 month before mobilization, and dosing within specified CD34+ cells/kg ranges.

"Metachromatic leukodystrophy (MLD) — Lenmeldy is indicated for the treatment of pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ), or early symptomatic early juvenile (ES..."