Kanuma® (sebelipase alfa) Injection

Kanuma (sebelipase alfa) is covered only for the FDA‑approved indication of lysosomal acid lipase (LAL) deficiency and is not authorized for non‑FDA indications. Approval (up to 12 months) requires confirmed diagnosis by deficient LAL enzyme activity in leukocytes, fibroblasts, or liver tissue or by molecular genetic testing showing an LAL gene mutation, prescription/consultation with a geneticist, endocrinologist, metabolic disorder subspecialist or other lysosomal storage disorder specialist, and age/presentation‑specific dosing (infants with rapidly progressive disease: start 1 mg/kg IV weekly, escalate to 3 then 5 mg/kg weekly if needed; pediatric/adult: 1 mg/kg IV every other week, escalate to 3 mg/kg every other week for suboptimal response).

"Kanuma (sebelipase alfa) is indicated for the treatment of individuals with lysosomal acid lipase (LAL) deficiency."