Gene Therapies for Metachromatic Leukodystrophy

This policy covers atidarsagene autotemcel (Lenmeldy), a lentiviral gene therapy for treatment of metachromatic leukodystrophy (MLD). Coverage is limited to pediatric patients with confirmed biallelic ARSA pathogenic/likely pathogenic variants or biochemical confirmation (low leukocyte ARSA and elevated urinary sulfatides) who are pre-symptomatic late infantile, pre-symptomatic early juvenile, or early symptomatic early juvenile, and who meet institutional stem cell transplant and organ-function requirements (adequate DLCO, LVEF, eGFR), have no prior allogeneic HSCT or prior gene therapy, and satisfy applicable FDA/compendia or peer‑review evidence and member benefit plan limitations.

"Coverage of any FDA‑approved drug when prescribed for a use recognized as safe and effective in one or more standard medical reference compendia adopted by the U."

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