BlueCross BlueShield of Tennessee Medical Policy Manual

Sebelipase Alfa

NDC CODE(S)

25682-0007-01 - Kanuma 20 mg/10 mL single-use vials (Alexion)

DESCRIPTION

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).

REFER TO DECISION SUPPORT TREE

POLICY

MEDICAL APPROPRIATENESS

INITIAL APPROVAL

RENEWAL CRITERIA

INDICATION(S)

DOSAGE & ADMINISTRATION

LAL Deficiency

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

APPLICABLE TENNESSEE STATE MANDATE REQUIREMENTS

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.

IMPORTANT REMINDER

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.

ADDITIONAL INFORMATION 

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).

No controlled studies were found in the published literature that validate the use of sebelipase alfa for the treatment of other conditions or diseases.

SOURCES

Lexi-Comp Online. (2016, October). AHFS DI. Sebelipase alfa. Retrieved November 23, 2016 from Lexi-Comp Online with AHFS.

MICROMEDEX Healthcare Series. Drugdex Drug Evaluations. (2016, September). Sebelipase alfa. Retrieved November 23, 2016 from MICROMEDEX Healthcare Series.

U. S. Food and Drug Administration. (2015, December). Center for Drug Evaluation and Research. Kanuma™ (sebelipase alfa). Retrieved November 23, 2016 from http://www.accessdata.fda.gov/drugsatfda_docs/label/2015/125561s000lbl.pdf.

ORIGINAL EFFECTIVE DATE:  1/6/2016

MOST RECENT REVIEW DATE:  2/27/2018

ID_MRx

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.

Pharmaceutical Decision Support Tree

Sebelipase Alfa (Kanuma ™)

  1. Is this the initial request for this agent?

If yes, go to question #2

If no, go to question #4

  1. Is the individual a month old with a 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?

If yes, go to question #3

If no, this does not meet medical necessity and/or medical appropriateness criteria

  1. Is the request for 340 billable units once weekly for dosage of 1 mg/kg administered once every other week as an IV infusion unless it is rapidly progressive disease presenting within the first 6 months of life when it is given 1 mg/kg administered once weekly as an IV infusion and it may increase to 3 mg/kg once weekly for patients who do not achieve an optimal clinical response for an authorization period of 6 months which may be renewed for a year?

If yes, this satisfies medical necessity and medical appropriateness criteria

If no, this does not meet medical necessity and/or medical appropriateness criteria

  1. Does the individual continue to meet the initial approval criteria in questions 2 and 3 with absence of unacceptable toxicity from the agent such as diarrhea, vomiting, fever, rhinitis, anemia, cough, nasopharyngitis, urticaria, headache, oropharyngeal pain, asthenia, constipation, nausea?

If yes, go to question #5

If no, this does not meet medical necessity and/or medical appropriateness criteria

  1. Has treatment resulted in clinical benefit, including, but not limited to, improvement in weight-for-age z-scores for patients exhibiting growth failure, improvement in LDL, HDL, OR triglycerides?

If yes, this satisfies medical necessity and medical appropriateness criteria

If no, this does not meet medical necessity and/or medical appropriateness criteria

This document has been classified as public information.