| Summary: | Sebelipase alfa (Kanuma) is a recombinant form of the human lysosomal acid lipase (LAL) enzyme, and is indicated for the treatment of infants, pediatric, and adult patients diagnosed with LAL deficiency. The recommended starting dose in infants (less than six months of age) presenting with rapidly progressive LAL deficiency is 1 mg/kg administered as an intravenous infusion once weekly, with dose escalation to 3 mg/kg once weekly, based on clinical response. In pediatric and adult patients the recommended dose is 1 mg/kg administered as an intravenous infusion once every other week. The manufacturer submitted a price of $8,546 per 10 mL vial of sebelipase alfa, containing 2 mg/mL concentrate solution for infusion. The average annual cost for sebelipase alfa in infantile-presentation patients ranges from $892,000 to $4.9 million per patient. In the pediatric/adult presentation, the average annual cost for sebelipase alfa is $892,000 per patient. The manufacturer submitted two cost-utility analyses based on the classification of LAL deficiency by infantile presentation and a pediatric/adult presentation
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