Lysosomal Storage Disorder Treatments

Aetna covers agalsidase beta (Fabrazyme) for confirmed Fabry disease and alglucosidase alfa (Lumizyme) for confirmed Pompe disease — including continuation for members responding to therapy — with Fabrazyme not to be used in combination with Galafold. Precertification (SMN) and adherence to the site‑of‑care policy are required for listed enzyme replacement therapies under commercial medical plans, and all other indications and specified interventions (e.g., concomitant use of certain glucocerebrosidase products, intrathecal idursulfase, gene therapy, pharmacological chaperones, substrate reduction) are considered experimental/investigational and excluded.

"Sebelipase alfa (Kanuma) — in infants with rapidly progressive disease starting dose and escalation guidance; labeling cautions regarding systemic hypersensitivity to eggs or egg products."

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