25682-0007-XX - Kanuma 20 MG/10 ML SOLN (ALEXION PHARMACEUTICALS)
Sebelipase alfa is a recombinant human lysosomal acid lipase (rhLAL). Lysosomal acid lipase (LAL) is a lysosomal glycoprotein enzyme that catalyzes the hydrolysis of cholesteryl esters to free cholesterol and fatty acids and the hydrolysis of triglycerides to glycerol and free fatty acids.
LAL deficiency is an autosomal recessive lysosomal storage disorder characterized by a genetic defect resulting in a marked decrease or loss in activity of the lysosomal acid lipase enzyme. The primary site of action of the LAL enzyme is the lysosome, where the enzyme normally causes the breakdown of lipid particles including LDL-c. Deficient LAL enzyme activity results in progressive complications due to the lysosomal accumulation of cholesteryl esters and triglycerides in multiple organs, including the liver, spleen, intestine, and the walls of blood vessels. The resulting lipid accumulation in the liver may lead to increased liver fat content and progression of liver disease, including fibrosis and cirrhosis. Lipid accumulation in the intestinal wall leads to malabsorption and growth failure. In parallel, dyslipidemia due to impaired degradation of lysosomal lipid is common with elevated LDL-c and triglycerides and low HDL-cholesterol (HDL-c).
Sebelipase alfa for the treatment of lysosomal acid lipase deficiency is considered medically necessary if the medical appropriateness criteria are met. (See Medical Appropriateness below.)
Sebelipase alfa for the treatment of other conditions/diseases is considered investigational.
Sebelipase alfa is considered medically appropriate if ALL of the following criteria are met:
Diagnosis of lysosomal acid lipase deficiency has been confirmed by either biallelic pathogenic variants in LIPA or deficient LAL enzyme activity in peripheral blood leukocytes, fibroblasts, or dried blood spots
Individual is at least one month old
Sebelipase alfa is considered medically appropriate for renewal if ALL of the following criteria are met:
Individual continues to meet initial approval criteria
Absence of unacceptable toxicity from the drug, e.g., hypersensitivity reactions including anaphylaxis, abdominal pain, fever, chills, pruritus, rash, vomiting, etc.
Treatment has resulted in clinical benefit, including, but not limited to:
Improvement in weight-for-age z-scores for individuals exhibiting growth failure
Improvement in LDL
Improvement in HDL
Improvement in triglycerides
Improvement in AST or ALT
DOSAGE & ADMINISTRATION
Pediatric & Adult patients:
§ 1 mg/kg administered once every other week as an IV infusion
Rapidly progressive disease presenting within the first 6 months of life:
§ 1 mg/kg administered once weekly as an IV infusion
May increase to 3 mg/kg once weekly for patients who do not achieve an optimal clinical response
LENGTH OF AUTHORIZATION
Coverage will be provided for six months and may be renewed annually thereafter
Refer to DOSAGE LIMITS below
BlueCross BlueShield of Tennessee’s Medical Policy complies with Tennessee Code Annotated Section 56-7-2352 regarding coverage of off-label indications of Food and Drug Administration (FDA) approved drugs when the off-label use is recognized in one of the statutorily recognized standard reference compendia or in the published peer-reviewed medical literature.
We develop Medical Policies to provide guidance to Members and Providers. This Medical Policy relates only to the services or supplies described in it. The existence of a Medical Policy is not an authorization, certification, explanation of benefits or a contract for the service (or supply) that is referenced in the Medical Policy. For a determination of the benefits that a Member is entitled to receive under his or her health plan, the Member's health plan must be reviewed. If there is a conflict between the Medical Policy and a health plan, the express terms of the health plan will govern.
For appropriate chemotherapy regimens, dosage information, contraindications, precautions, warnings, and monitoring information, please refer to one of the standard reference compendia (e.g., the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) published by the National Comprehensive Cancer Network®, Drugdex Evaluations of Micromedex Solutions at Truven Health, or The American Hospital Formulary Service Drug Information).
Lexi-Comp Online. (2019, February). AHFS DI. Sebelipase alfa. Retrieved February 22, 2019 from Lexi-Comp Online with AHFS.
MICROMEDEX Healthcare Series. Drugdex Drug Evaluations. (2018, April). Sebelipase alfa. Retrieved February 22, 2019 from MICROMEDEX Healthcare Series.
U. S. Food and Drug Administration. (2015, December). Center for Drug Evaluation and Research. Kanuma® (sebelipase alfa). Retrieved February 22, 2019 from https://www.accessdata.fda.gov/drugsatfda_docs/label/2015/125561s000lbl.pdf.
ORIGINAL EFFECTIVE DATE: 1/6/2016
MOST RECENT REVIEW DATE: 4/9/2019
Policies included in the Medical Policy Manual are not intended to certify coverage availability. They are medical determinations about a particular technology, service, drug, etc. While a policy or technology may be medically necessary, it could be excluded in a member's benefit plan. Please check with the appropriate claims department to determine if the service in question is a covered service under a particular benefit plan. Use of the Medical Policy Manual is not intended to replace independent medical judgment for treatment of individuals. The content on this Web site is not intended to be a substitute for professional medical advice in any way. Always seek the advice of your physician or other qualified health care provider if you have questions regarding a medical condition or treatment.
This document has been classified as public information.
Maximum billable units per dose and over time by indication as a Medical Benefit; 1 billable unit = 1 mg