Notification



Notification Issue Date:



Medicare Advantage Policy

Title:Sebelipase alfa (Kanuma®)
Policy #:MA08.078c

This policy is applicable to the Company’s Medicare Advantage products only. Policies that are applicable to the Company’s commercial products are accessible via a separate commercial policy database.


The Company makes decisions on coverage based on the Centers for Medicare and Medicaid Services (CMS) regulations and guidance, benefit plan documents and contracts, and the member’s medical history and condition. If CMS does not have a position addressing a service, the Company makes decisions based on Company Policy Bulletins. Benefits may vary based on contract, and individual member benefits must be verified. The Company determines medical necessity only if the benefit exists and no contract exclusions are applicable. Although the Medicare Advantage Policy Bulletin is consistent with Medicare’s regulations and guidance, the Company’s payment methodology may differ from Medicare.

When services can be administered in various settings, the Company reserves the right to reimburse only those services that are furnished in the most appropriate and cost-effective setting that is appropriate to the member’s medical needs and condition. This decision is based on the member’s current medical condition and any required monitoring or additional services that may coincide with the delivery of this service.


This Policy Bulletin document describes the status of CMS coverage, medical terminology, and/or benefit plan documents and contracts at the time the document was developed. This Policy Bulletin will be reviewed regularly and be updated as Medicare changes their regulations and guidance, scientific and medical literature becomes available, and/or the benefit plan documents and/or contracts are changed.



Policy

Coverage is subject to the terms, conditions, and limitations of the member's Evidence of Coverage.

MEDICALLY NECESSARY

Sebelipase alfa (Kanuma®) is considered medically necessary and, therefore, covered for individuals with lysosomal acid lipase (LAL) deficiency when the following criteria, including Dosing and Frequency Requirements, are met:
  • Diagnosis of LAL deficiency is confirmed by either of the following:
    • Confirmation of the absence or deficiency of LAL resulting from dried blood spot (DBS) testing
    • Confirmation of molecular genetic testing that reveals pathogenic variation(s) in the lipase A, lysosomal acid type (LIPA) gene, which results in reduction of functionality of LAL enzyme activity
  • Documented presence of clinical signs and symptoms of the disease (e.g., hepatomegaly, dyslipidemia, increased serum transaminases [ALT and AST], liver cirrhosis/fibrosis)

CONTINUATION THERAPY
Sebelipase alfa (Kanuma®) is considered medically necessary and, therefore, covered for continuation therapy of LAL deficiency in individuals who have continued improvement in their disease (e.g., improvement in symptoms, lipid panels, serum transaminases).

DOSING AND FREQUENCY REQUIREMENTS

The following dosage and frequency information was taken from the Prescribing Information for this product:
  • Infants with rapidly progressive LAL deficiency presenting within the first 6 months of life: 1 mg/kg I.V. once weekly. If optimal clinical response is not achieved, increase to 3 mg/kg once weekly.
  • Pediatric and Adult Individuals with LAL Deficiency: 1 mg/kg I.V. once every other week.

The Company reserves the right to modify the Dosing and Frequency Requirements listed in this policy to ensure consistency with the most recently published recommendations for the use of sebelipase alfa (Kanuma®). Changes to these guidelines are based on a consensus of information obtained from resources such as, but not limited to: the US Food and Drug Administration (FDA); Company-recognized authoritative pharmacology compendia; or published peer-reviewed clinical research. The professional provider must supply supporting documentation (i.e., published peer-reviewed literature) in order to request coverage for an amount of sebelipase alfa (Kanuma®) outside of the Dosing and Frequency Requirements listed in this policy. For a list of Company-recognized pharmacology compendia, view our policy on off-label coverage for prescription drugs and biologics.

Accurate member information is necessary for the Company to approve the requested dose and frequency of this drug. If the member’s dose, frequency, or regimen changes (based on factors such as changes in member weight or incomplete therapeutic response), the provider must submit those changes to the Company for a new approval based on those changes as part of the utilization management activities. The Company reserves the right to conduct post-payment review and audit procedures for any claims submitted for sebelipase alfa (Kanuma®).

EXPERIMENTAL/INVESTIGATIONAL

All other uses for sebelipase alfa (Kanuma®) are considered experimental/investigational and, therefore, not covered unless the indication is supported as an accepted off-label use, as defined in the Company medical policy on off-label coverage for prescription drugs and biologics.

REQUIRED DOCUMENTATION

The individual's medical record must reflect the medical necessity for the care provided. These medical records may include, but are not limited to: records from the professional provider's office, hospital, nursing home, home health agencies, therapies, and test reports.

The Company may conduct reviews and audits of services to our members, regardless of the participation status of the provider. All documentation is to be available to the Company upon request. Failure to produce the requested information may result in a denial for the drug.

When coverage of sebelipase alfa (Kanuma®) is requested outside of the Dosing and Frequency Requirements listed in this policy, the prescribing professional provider must supply documentation (i.e., published peer-reviewed literature) to the Company that supports this request.
Policy Guidelines

BENEFIT APPLICATION

Subject to the terms and conditions of the applicable Evidence of Coverage, sebelipase alfa (Kanuma®) is covered under the medical benefits of the Company’s Medicare Advantage products when the medical necessity criteria and Dosing and Frequency Requirements listed in this medical policy are met.

For Medicare Advantage members, certain drugs are available through either the member's medical benefit (Part B benefit) or pharmacy benefit (Part D benefit), depending on how the drug is prescribed, dispensed, or administered. This policy only addresses instances when sebelipase alfa (Kanuma®) is covered under a member's medical benefit (Part B benefit). It does not address instances when sebelipase alfa (Kanuma®) is covered under a member’s pharmacy benefit (Part D benefit).

HOME INFUSION

Sebelipase alfa (Kanuma®) may be available under the medical benefit for applicable lines of business through home infusion providers. Member’s benefit for home infusion should be verified.

US FOOD AND DRUG ADMINISTRATION (FDA) STATUS

Sebelipase alfa (Kanuma®) was approved by the FDA on December 8, 2015 for individuals with lysosomal acid lipase deficiency. The safety and effectiveness of sebelipase alfa (Kanuma®) have been established in pediatric individuals aged 1 month and older.

Description

Lysosomal acid lipase deficiency is an autosomal recessive lysosomal storage disease characterized by a genetic defect in the LIPA gene resulting in a marked decrease or loss of activity of the lysosomal acid lipase (LAL) enzyme. LAL breaks down lipid to become free cholesterol and free fatty acid. Free cholesterol and free fatty acids act as transcription factors to down regulate the synthesis and uptake of cholesterol and lipogenesis. Deficient LAL enzyme results in progressive complications due to lysosomal accumulation of cholesteryl esters and triglycerides in multiple organs, including the liver, spleen, intestine, and the walls of the blood vessels.

The most common LIPA gene mutation, found in about half of individuals with LAL deficiency that begins in childhood or later, is called a splice-site mutation where there is a substitution of guanine for adenine near an area of the gene called exon 8 (IVS8-1G>A).

Early-onset LAL deficiency, also known as Wolman disease, typically presents within the first weeks of life, and affected infants usually die within 6-12 months. The common symptoms for the disease in infants are prominent hepatosplenomegaly, diarrhea and vomiting, malabsorption, growth failure, and liver failure. Late-onset LAL deficiency, also known as cholesteryl ester storage disease, has a mean age of onset of 5 years with the primary symptom of hepatomegaly.

Sebelipase alfa (Kanuma®) was approved by the FDA on December 8, 2015 for the treatment of individuals with a diagnosis of lysosomal acid lipase deficiency. Sebelipase alfa (Kanuma®) is a recombinant human lysosomal acid lipase catalyzing the hydrolysis of cholesteryl ester to free cholesterol and free fatty acids.

CLINICAL STUDIES

A multicenter, open-label, single-arm clinical study on the efficacy of sebelipase alfa (Kanuma®) was conducted in 9 infants with LAL deficiency who had growth failure or other rapidly progressive disease prior to 6 months of age. Efficacy was assessed by comparing the survival of infants treated with sebelipase alfa (Kanuma®) to 21 historical cohort infants with a similar age at disease presentation and clinical characteristics who were not treated with sebelipase alfa (Kanuma®). Of the 9 infants on sebelipase alfa (Kanuma®) 6 survived beyond 12 months of age compared to the historical cohort infants with 0 surviving past 12 months, all of whom died by 8 months of age. The median age of the 6 infants who survived beyond 12 months was 18.1 months.

The safety and efficacy of sebelipase alfa (Kanuma®) were assessed in 66 pediatric and adult individuals with late-onset LAL deficiency, age range 4 to 58 years, in a multicenter, double-blind, placebo-controlled trial. At the completion of the 20-week double-blind period, the sebelipase alfa (Kanuma®)--treated group had a statistically significant improvement in percent change from baseline in LDL-c, non-HDL-c, triglycerides, and HDL-c than the placebo group. Individuals treated with sebelipase alfa (Kanuma®) had a larger reduction from baseline in ALT values and liver fat content compared to individuals treated with placebos.

OFF-LABEL INDICATIONS

There may be additional indications contained in the Policy section of this document due to evaluation of criteria highlighted in the Company’s off-label policy, and/or review of clinical guidelines issued by leading professional organizations and government entities.
References

American Hospital Formulary Service (AHFS). Drug Information 2018. Sebelipase alfa. [Lexicomp Online Web site]. 03/22/2017. Available at: http://online.lexi.com/lco/action/home[via subscription only]. Accessed July 23, 2018.

Camarena C, Aldamiz-Echevarria LJ, Polo B, et al. Update on lysosomal acid lipase deficiency: Diagnosis, treatment and patient management. Med Clin (Barc). 2017 May 10;148(9):429.e1-429.e10.

Elsevier’s Clinical Pharmacology Compendium. Sebelipase alfa. [ClinicalKey Web site]. 01/06/2016. Available at: https://www.clinicalkey.com/pharmacology/ [via subscription only]. Accessed July 23, 2018.

Lexi-Drugs Compendium. Sebelipase alfa. [Lexicomp Online Web site]. 07/07/2018. Available at: http://online.lexi.com/lco/action/home [via subscription only]. Accessed July 23, 2018.

Micromedex® Healthcare Series [Internet database]. DRUGDEX® Evaluations. Sebelipase alfa (Kanuma®). Greenwood Village, CO: Thomson Micromedex. 04/30/2018. Available at:
http://www.micromedexsolutions.com/micromedex2/librarian. Accessed July 23, 2018.

National Institutes of Health (NIH). Genetics Home Reference. LIPA Gene. Reviewed 02/2017. Available at: https://ghr.nlm.nih.gov/gene/LIPA#conditions . Accessed August 16, 2018.

Reiner Z, Guardamagna O, Nair D, etc. Lysosmal acid lipase deficiency: An under-recognized cause of dyslipidaemia and liver dysfunction. Atherosclerosis.2014; 235:21-30

US Food and Drug Administration. Sebelipase alfa. Product Information. 12/2015. Available at: https://www.accessdata.fda.gov/scripts/cder/daf/ or http://www.accessdata.fda.gov/drugsatfda_docs/label/2015/125561s000lbl.pdf. Accessed July 20, 2018.



Coding

Inclusion of a code in this table does not imply reimbursement. Eligibility, benefits, limitations, exclusions, precertification/referral requirements, provider contracts, and Company policies apply.

The codes listed below are updated on a regular basis, in accordance with nationally accepted coding guidelines. Therefore, this policy applies to any and all future applicable coding changes, revisions, or updates.

In order to ensure optimal reimbursement, all health care services, devices, and pharmaceuticals should be reported using the billing codes and modifiers that most accurately represent the services rendered, unless otherwise directed by the Company.

The Coding Table lists any CPT, ICD-9, ICD-10, and HCPCS billing codes related only to the specific policy in which they appear.

CPT Procedure Code Number(s)

N/A


Professional and outpatient claims with a date of service on or before September 30, 2015, must be billed using ICD-9 codes. Professional and outpatient claims with a date of service on or after October 1, 2015, must be billed using ICD-10 codes.

Facility/Institutional inpatient claims with a date of discharge on or before September 30, 2015, must be billed with ICD-9 codes. Facility/Institutional inpatient claims with a date of discharge on or after October 1, 2015, must be billed with ICD-10 codes.


ICD - 10 Procedure Code Number(s)

N/A


Professional and outpatient claims with a date of service on or before September 30, 2015, must be billed using ICD-9 codes. Professional and outpatient claims with a date of service on or after October 1, 2015, must be billed using ICD-10 codes.

Facility/Institutional inpatient claims with a date of discharge on or before September 30, 2015, must be billed with ICD-9 codes. Facility/Institutional inpatient claims with a date of discharge on or after October 1, 2015, must be billed with ICD-10 codes.


ICD -10 Diagnosis Code Number(s)

E75.5 Other lipid storage disorders


HCPCS Level II Code Number(s)

J2840 Injection, sebelipase alfa, 1 mg


Revenue Code Number(s)

N/A

Coding and Billing Requirements


Cross References




Policy History

Revisions for MA08.078c
09/25/2019This policy has been reissued in accordance with the Company's annual review process.
12/03/2018This version of the policy will become effective 12/03/2018.

This policy was updated to include additional coverage criteria, including diagnostic testing, continuation therapy, and Dosage and Frequency.

Revisions for MA08.078b
06/06/2018This policy has been reissued in accordance with the Company's annual review process.
06/07/2017The policy has been reviewed and reissued to communicate the Company’s continuing position on sebelipase alfa (Kanuma).
01/01/2017This policy has been identified for the HCPCS code update, effective 01/01/2017.

The following HCPCS code has been termed from this policy:
C9478 Injection, sebelipase alfa, 1 mg

The following NOC code has been removed from this policy and is replaced by the following HCPCS code:

REMOVED: J3590 Unclassified biologics
REPLACED WITH: J2840 Injection, sebelipase alfa, 1 mg

Revisions for MA08.078a
07/01/2016This policy has been identified for the HCPCS code update, effective 07/01/2016.

The following NOC code has been removed from this policy and is replaced by the following HCPCS code:

REMOVED: C9399 Unclassified drugs or biologicals
REPLACED WITH: C9478 Injection, sebelipase alfa, 1 mg

Revisions for MA08.078
02/24/2016This new policy has been developed to communicate of the Company’s coverage criteria for sebelipase alfa (Kanuma®).




Version Effective Date: 12/03/2018
Version Issued Date: 12/03/2018
Version Reissued Date: 09/26/2019