Neurology - Lenmeldy™

Lenmeldy is covered as a one-time, per‑lifetime IV gene therapy only for children with specified early‑onset MLD subtypes (PSLI, PSEJ, ESEJ) who meet required ARSA genotype, low ARSA enzyme activity, elevated 24‑hour urine sulfatide, and clinical-status criteria (presymptomatic PSLI/PSEJ or ESEJ with independent walking GMFC‑MLD 0–1 and IQ ≥85). Approval requires extensive documentation and verification (genotype, labs, negative infectious screening, prescriber specialty—hematologist/neurologist/medical geneticist/stem‑cell transplant specialist), planned HSCT with mobilization/apheresis and myeloablative conditioning including G‑CSF ± mobilizer and busulfan, adherence to subtype‑specific CD34+ cells/kg dosing ranges, weight within 30 days, administration within a 1‑year approval, and excludes late‑juvenile/adult MLD, GMFC‑MLD >1, prior Lenmeldy or prior gene therapy, and recent allogeneic HSCT within 6 months or residual donor cells.

"Treatment of metachromatic leukodystrophy (MLD) in children who meet ALL FDA-approved criteria below (one-time, per lifetime, single intravenous infusion)."