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Horizon BCBSNJ
Uniform Medical Policy ManualSection:Drugs
Policy Number:136
Effective Date: 09/11/2020
Original Policy Date:01/26/2016
Last Review Date:09/08/2020
Date Published to Web: 01/26/2016
Subject:
Sebelipase alfa (Kanuma)

Description:
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IMPORTANT NOTE:

The purpose of this policy is to provide general information applicable to the administration of health benefits that Horizon Blue Cross Blue Shield of New Jersey and Horizon Healthcare of New Jersey, Inc. (collectively “Horizon BCBSNJ”) insures or administers. If the member’s contract benefits differ from the medical policy, the contract prevails. Although a service, supply or procedure may be medically necessary, it may be subject to limitations and/or exclusions under a member’s benefit plan. If a service, supply or procedure is not covered and the member proceeds to obtain the service, supply or procedure, the member may be responsible for the cost. Decisions regarding treatment and treatment plans are the responsibility of the physician. This policy is not intended to direct the course of clinical care a physician provides to a member, and it does not replace a physician’s independent professional clinical judgment or duty to exercise special knowledge and skill in the treatment of Horizon BCBSNJ members. Horizon BCBSNJ is not responsible for, does not provide, and does not hold itself out as a provider of medical care. The physician remains responsible for the quality and type of health care services provided to a Horizon BCBSNJ member.

Horizon BCBSNJ medical policies do not constitute medical advice, authorization, certification, approval, explanation of benefits, offer of coverage, contract or guarantee of payment.

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Kanuma (sebelipase alfa) is a hydrolytic lysosomal cholesteryl ester and triacylglycerol-specific enzyme indicated for the treatment of patients with a diagnosis of Lysosomal Acid Lipase (LAL) deficiency (also known as Wolman disease and cholesteryl ester storage disease [CESD]). Kanuma is the first FDA approved medication for this indication. The FDA granted Kanuma orphan drug designation because it treats a rare disease affecting fewer than 200,000 patients in the United States.

The safety and efficacy of Kanuma were tested in an open-label, historically controlled trial in nine infants with rapidly progressive Wolman disease and in a double-blind, placebo-controlled trial in 66 pediatric and adult patients with CESD. In the trial in infants with Wolman disease, six of nine infants (67 percent) treated with Kanuma were alive at 12 months of age, whereas none of the 21 infants in the historical control group survived. In the trial in CESD patients, there was a statistically significant improvement in LDL-cholesterol levels and other disease-related parameters in those treated with Kanuma versus placebo after 20 weeks of treatment.

The most common side effects observed in patients treated with Kanuma are diarrhea, vomiting, fever, rhinitis, anemia, cough, headache, constipation, and nausea. Additional safety concerns include hypersensitivity reactions and use in individuals with hypersensitivity to eggs or egg products.

Policy:
(NOTE: For Medicare Advantage, please refer to the Medicare Coverage Section below for coverage guidance)

The requirements of the Horizon BCBSNJ Sebelipase alfa (Kanuma) Program may require a precertification/prior authorization via MagellanRx Management. These requirements are member-specific: please verify member eligibility and requirements through the Horizon Provider Portal (www.horizonblue.com/provider). Ordering clinicians should request pre-certification from MagellanRx Management at ih.magellanrx.com or call 1-800-424-4508 (when applicable).

1. Kanuma (sebelipase alfa) is medically necessary for the treatment of Lysosomal Acid Lipase (LAL) deficiency in members at least 1 month old who have diagnosis of lysosomal acid lipase deficiency (LAL-D) with one of the following:

    a. diagnosis documented by genetic test revealing mutations in the lipase A, lysosomal acid type (LIPA) gene (documentation of medical records required), OR
    b. diagnosis documented by dried blood spot test or leukocyte or fibroblast test demonstrating deficient lysosomal acid lipase activity (documentation of medical records required)

2. When Kanuma (sebelipase alfa) is medically necessary, therapy will be approved for 6 months based on FDA recommendations:
    a. LAL deficiency presenting within the first 6 months of life: 1 mg/kg as an intravenous infusion once weekly (may increase to 3 mg/kg once weekly for non-optimal clinical response)
    b. Pediatric and adult patients with LAL deficiency: 1 mg/kg as an intravenous infusion once every other week
    c. Interrupt Kanuma if a severe hypersensitivity reaction occurs

3. Continued therapy with Kanuma (sebelipase alfa) will be approved every 12 months based on the following treatment response (documentation of medical records required) and absence of intolerable adverse effects.
            • Improvement in weight-for-age z-scores for patients exhibiting growth failure
            • Improvement in LDL
            • Improvement in HDL
            • Improvement in triglycerides
            • Improvement of AST or ALT

4. Kanuma (sebelipase alfa) for the treatment of other conditions/diseases is considered investigational.

Medicare Coverage

There is no National Coverage Determination (NCD). In the absence of an NCD, coverage decisions are left to the discretion of Local Medicare Carriers. Novitas Solutions, Inc, the Local Medicare Carrier for jurisdiction JL, has not issued a determination for this service. Therefore, Medicare Advantage Products will follow the Horizon Policy.

Medicaid Coverage

For Horizon NJ Health members, please follow this link for the corresponding HNJH drug policy https://services3.horizon-bcbsnj.com/ddn/NJhealthWeb.nsf

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Horizon BCBSNJ Medical Policy Development Process:

This Horizon BCBSNJ Medical Policy (the “Medical Policy”) has been developed by Horizon BCBSNJ’s Medical Policy Committee (the “Committee”) consistent with generally accepted standards of medical practice, and reflects Horizon BCBSNJ’s view of the subject health care services, supplies or procedures, and in what circumstances they are deemed to be medically necessary or experimental/ investigational in nature. This Medical Policy also considers whether and to what degree the subject health care services, supplies or procedures are clinically appropriate, in terms of type, frequency, extent, site and duration and if they are considered effective for the illnesses, injuries or diseases discussed. Where relevant, this Medical Policy considers whether the subject health care services, supplies or procedures are being requested primarily for the convenience of the covered person or the health care provider. It may also consider whether the services, supplies or procedures are more costly than an alternative service or sequence of services, supplies or procedures that are at least as likely to produce equivalent therapeutic or diagnostic results as to the diagnosis or treatment of the relevant illness, injury or disease. In reaching its conclusion regarding what it considers to be the generally accepted standards of medical practice, the Committee reviews and considers the following: all credible scientific evidence published in peer-reviewed medical literature generally recognized by the relevant medical community, physician and health care provider specialty society recommendations, the views of physicians and health care providers practicing in relevant clinical areas (including, but not limited to, the prevailing opinion within the appropriate specialty) and any other relevant factor as determined by applicable State and Federal laws and regulations.

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Index:
Sebelipase alfa (Kanuma)
Kanuma (Sebelipase alfa)

References:
1. Kanuma® [package insert]. Alexion Pharmaceuticals, Inc. Cheshire, CT. December 2015.
2. FDA News Release: FDA approves first drug to treat a rare enzyme disorder in pediatric and adult patients. Available at: http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm476013.htm

3. Feillet F, et. al. A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency. N Engl J Med 2015; 373:1010-1020September 10, 2015DOI: 10.1056/NEJMoa1501365

4. ClinicalTrials.gov. Available at: https://clinicaltrials.gov/ct2/results?term= Kanuma=Search. Accessed March 2020.

5. Hoffman E, Barr M, Giovanni M, Murray M. Lysosomal Acid Lipase Deficiency. GeneReviews® [Internet]. Available at: www.ncbi.nlm.nih.gov/books/NBK305870/

6. Hamilton J, Jones I, Srivastava R. A new method for the measurement of lysosomal acid lipase in dried blood spots using the inhibitor Lalistat 2. Clin Chim Acta. 2012 Aug 16;413(15-16):1207-10. doi: 10.1016/j.cca.2012.03.019.

Codes:
(The list of codes is not intended to be all-inclusive and is included below for informational purposes only. Inclusion or exclusion of a procedure, diagnosis, drug or device code(s) does not constitute or imply authorization, certification, approval, offer of coverage or guarantee of payment.)

CPT*

    HCPCS
    J2840


    * CPT only copyright 2020 American Medical Association. All rights reserved. CPT is a registered trademark of the American Medical Association.

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    Medical policies can be highly technical and are designed for use by the Horizon BCBSNJ professional staff in making coverage determinations. Members referring to this policy should discuss it with their treating physician, and should refer to their specific benefit plan for the terms, conditions, limitations and exclusions of their coverage.

    The Horizon BCBSNJ Medical Policy Manual is proprietary. It is to be used only as authorized by Horizon BCBSNJ and its affiliates. The contents of this Medical Policy are not to be copied, reproduced or circulated to other parties without the express written consent of Horizon BCBSNJ. The contents of this Medical Policy may be updated or changed without notice, unless otherwise required by law and/or regulation. However, benefit determinations are made in the context of medical policies existing at the time of the decision and are not subject to later revision as the result of a change in medical policy

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