CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Sebelipase alfa (Kanuma) — Coverage Criteria for Lysosomal Acid Lipase (LAL) Deficiency

Covers medical benefit authorization, dosing, and renewal criteria for Kanuma (sebelipase alfa) for members with confirmed LAL deficiency across commercial, Medicaid, and Medicare‑Medicaid Plan benefits as specified.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicySebelipase alfa (Kanuma) — Coverage Criteria for LAL deficiency

Policy CodePolicy N/A

Change TypeNo material change

Effective DateDec 20, 2017

Next Review DateN/A

Key ActionObtain prior authorization with documentation of patient age, prescribing specialist (genetics/metabolism), weight, baseline liver function and lipid panel, and confirmation of diagnosis via biallelic LIPA variants or deficient LAL enzyme activity.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

6 monthsLength of auth

1→5 mg/kgDose escalation

J2840HCPCS

E75.5ICD-10

112 vials/28dQuantity limit

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Coverage Criteria for Sebelipase alfa (Kanuma)

inv-01: Initial Approval — Covered when ALL of the following are met

Covered when ALL of the following are met

Initial Approval Criteria: Patient is at least 1 month of age; prescribing physician is a specialist in genetics and metabolism; weight, baseline liver function and baseline lipid panel are provided; diagnosis of Lysosomal Acid Lipase (LAL) deficiency is confirmed by either biallelic pathogenic variants in LIPA or deficient LAL enzyme activity in peripheral blood leukocytes, fibroblasts, or dried blood spots.N/A

Members previously treated within the past 365 days are exempt from step therapy requirements.

inv-02: Renewal Criteria — Renewal is allowed when ANY of the following clinical conditions are met (with absence of unacceptable toxicity)

Renewal is allowed when ANY of the following clinical conditions are met (with absence of unacceptable toxicity)

Renewal Criteria - Clinical Benefit: Treatment has resulted in clinical benefit as evidenced by improvement in weight-for-age z-scores for growth failure, improvement in LDL, improvement in HDL, improvement in triglycerides, or improvement of AST or ALT.Documented improvement in one or more parameters

Absence of unacceptable toxicity required (e.g., no hypersensitivity or anaphylaxis).

Renewal Criteria - Dose Escalation (pediatric/adult): Dose escalation is warranted for pediatric and adult patients with a suboptimal clinical response to the 1 mg/kg dose, defined by at least one of: poor growth; deteriorating biochemical markers (e.g., ALT, AST); and/or adverse lipid parameters (e.g., LDL-c, triglycerides).Clinical evidence of suboptimal response

Supports escalation per dosing guidance.

Renewal Criteria - Infants with rapidly progressive disease: Dose escalation for infants presenting within the first 6 months of life who have a suboptimal clinical response to the 1 mg/kg or 3 mg/kg dose, defined by at least one of: poor growth; deteriorating biochemical markers (e.g., ALT, AST); or persistent or worsening organomegaly.Clinical evidence as listed

Allows escalation up to specified infant dosing.

These coverage criteria apply to Kanuma (sebelipase alfa) when there are no applicable National Coverage Determinations (NCDs) or Local Coverage Determinations (LCDs) that would govern coverage for INTEGRITY (Medicare‑Medicaid Plan) members. Neighborhood Health Plan of Rhode Island will consider requests individually using information submitted by the prescriber and other plan resources; the plan’s criteria are intended to align with FDA labeling and relevant clinical literature.

Use of sebelipase alfa is not supported when there is unacceptable toxicity related to the drug (examples include hypersensitivity reactions such as anaphylaxis). Renewal of authorization requires absence of unacceptable toxicity and evidence of clinical benefit as specified below; if renewal criteria are not met or unacceptable toxicity is present, continued coverage is not supported.

Billing, Codes, and Quantity Limits

Covered HCPCS CodesHCPCSCovered

J2840

Injection, sebelipase alfa, 1 mg

NDCsNDCCovered

25682-0007-xx

Kanuma 20 mg/10 mL single-dose vials (NDC)

Covered Diagnosis Codes (ICD-10)ICD-10Covered

E75.5

Other lipid storage disorders

inv-08: Quantity limit (vials) — 112 vials per 28 day supply

Quantity limit (vials)Kanuma 20 mg/10 mL single‑dose vials: 112 vials per 28 day supply

Supply intervalPer 28 day supply

NDC packaging referencedKanuma 20 mg/10 mL single‑dose vials

inv-09: HCPCS billing units — 560 billable units once weekly

HCPCS billing units560 billable units once weekly

Unit definition1 billable unit = 1 mg (J2840)

Prior Authorization, Documentation, and Provider Requirements

Prior Authorization

Prior Authorization Required

Prior authorization required. Approvals are issued for 6 months. Members who have received the medication within the past 365 days are not subject to step therapy requirements and may be eligible for authorization without prior prerequisite therapy.

Initial and renewal approvals: 6 months
Members treated within past 365 days bypass step therapy

Step Therapy

Step Therapy Exception

MMP members treated within the past 365 days bypass step therapy requirements and may proceed to authorization based on indication-specific criteria.

Applies to members with documented treatment within past 365 days

Background and Clinical Context

Lysosomal Acid Lipase (LAL) deficiency is a rare, inherited lipid storage disorder treated with enzyme replacement therapy using sebelipase alfa (Kanuma). Neighborhood’s coverage criteria were developed by the Pharmacy & Therapeutics Committee to align with FDA labeling and clinical evidence and to ensure use for medically accepted indications while minimizing adverse effects and unnecessary cost. Renewal guidance requires demonstrated clinical benefit — for example, improved weight‑for‑age z‑scores in patients with growth failure or improvements in lipid parameters (LDL, HDL, triglycerides) or liver enzymes (AST, ALT) — or justified dose escalation for suboptimal response, particularly in infants with rapidly progressive disease.

Definitions and Diagnostic Confirmation

inv-15: Confirmed LAL deficiency — diagnostic criteria (biallelic LIPA variants or enzyme assay)

Confirmatory genetic testingIdentification of biallelic pathogenic variants in the LIPA gene

Enzyme assay optionsDemonstration of deficient LAL enzyme activity in peripheral blood leukocytes

Alternative specimen types for enzyme testingDeficient LAL enzyme activity in fibroblasts or dried blood spots