Enzyme-Replacement Therapy for Lysosomal Storage Disorders

This policy covers enzyme‑replacement therapy (ERT) for lysosomal storage disorders, including FDA‑approved ERTs such as Cerezyme, Elelyso, and Vpriv, for conditions like Gaucher disease (including Type 1), mucopolysaccharidoses (MPS I, II, VI), acid sphingomyelinase deficiency/Niemann‑Pick disease (ASMD Types A/B/intermediate), and other lysosomal disorders causing neuromuscular, neurologic, or cognitive impairment. Coverage is subject to the member’s benefit plan and clinical criteria—FDA approval or compendia support required, off‑label use requires two peer‑reviewed articles, Vpriv is preferred for initial Gaucher Type 1 treatment, continuation requires documented clinical benefit, non‑preferred agents require documented failure/intolerance/contraindication to preferred agents, and state‑ or plan‑specific limitations (including pediatric and fully insured plan restrictions) apply.

"Coverage for therapy, diagnostic testing, and equipment necessary to increase quality of life for children clinically or genetically diagnosed with any disease, syndrome, or disorder that includes ..."