Sebelipase Alfa Injections (Kanuma®)

Sebelipase alfa (Kanuma) is covered only for the FDA‑approved treatment of lysosomal acid lipase (LAL) deficiency (not for non‑FDA uses unless separately authorized) and requires confirmation by deficient LAL enzyme activity in leukocytes, fibroblasts, or liver tissue or by molecular genetic testing plus prescription by or consultation with a geneticist, endocrinologist, metabolic disorder subspecialist, or lysosomal storage disorder specialist. Approval is for 12 months with IV dosing: infants with rapidly progressive disease start 1 mg/kg weekly (may escalate to 3 mg/kg then 5 mg/kg for suboptimal response) and pediatric/adults start 1 mg/kg every other week (may escalate to 3 mg/kg), with documentation of clinical response required to justify dose escalation.

"Treatment of individuals with lysosomal acid lipase (LAL) deficiency."

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