CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Kanuma

Defines coverage, dosing, renewal criteria, authorization length, quantity and billing limits, and required diagnostic confirmation for Kanuma (sebelipase alfa) for treatment of Lysosomal Acid Lipase (LAL) deficiency across Medicaid, Commercial, and Medicare medical benefit scopes.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyKanuma

Policy CodePolicy N/A

Change TypeNo material change

Effective Date

Next Review Date

Key ActionPrescribing physician must be a specialist in genetics and metabolism and provide weight, baseline liver function and baseline lipid panel for prior authorization and renewal documentation.

SourceLink

POLICY UPDATE CHANGES

No material clinical/coverage changes

1Covered ICD-10 diagnosis codes

6 monthsAuthorization length (initial/renewal)

112 vials/28dNDC quantity limit

560 units/weekHCPCS max units

Coverage Summary

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Coverage stance: covered_with_criteria. Scope: defines coverage across Medicaid, Commercial, and Medicare medical benefit scopes for treatment of Lysosomal Acid Lipase (LAL) deficiency. Policy subject: Kanuma (sebelipase alfa), an enzyme replacement therapy approved for the treatment of LAL deficiency (FDA-approved indication). The drug has orphan drug designation. Coverage is provided when the specified eligibility criteria are met (including age, specialist prescriber, diagnostic confirmation, and required baseline data) and billing is performed using the listed HCPCS and NDC codes.

Continuation / Renewal Criteria

Documentation Required

Renewal documentation of clinical benefit or toxicity absence

For renewal, the provider must document continued indication eligibility, absence of unacceptable toxicity, and evidence of clinical benefit or provide justification for dose escalation.

Examples of acceptable clinical benefit: improvement in growth (weight-for-age z-scores)
Improvement in LDL
Improvement in HDL
Improvement in triglycerides (TG)
Improvement in AST or ALT
If escalating dose, provider must justify the need for dose escalation based on suboptimal clinical response

Applicable Codes

HCPCS (covered billing code)HCPCSCovered

J2840

Injection, sebelipase alfa, 1 mg (1 billable unit = 1 mg)

NDCs (product availability / quantity limit)NDCCovered

25682-0007-xx

Kanuma 20 mg/10 mL single-dose vials

Covered Diagnosis CodesICD-10Covered

E75.5

Other lipid storage disorders

Billing Rule

HCPCS billing unit

Bill using HCPCS code J2840 where 1 billable unit = 1 mg of sebelipase alfa. Follow the maximum units limit of 560 billable units once weekly. NDC product availability: Kanuma 20 mg/10 mL single-dose vials (25682-0007-xx) with NDC quantity limits (112 vials per 28-day supply).

Provider Actions / Prior Authorization

Prior Authorization

Specialist prescriber requirement and documentation

Prescribing physician must be a specialist in genetics and metabolism. Provider must supply patient weight, baseline liver function tests, and a baseline lipid panel as part of the prior authorization request.

Prior Authorization

Diagnostic confirmation required

Document diagnostic confirmation by either: (1) biallelic pathogenic variants in LIPA; OR (2) deficient LAL enzyme activity measured in peripheral blood leukocytes, fibroblasts, or dried blood spots. Coverage applies for the covered diagnosis code E75.5 (Other lipid storage disorders).

Billing Rule

HCPCS billing unit

Documentation Required

Renewal documentation of clinical benefit or toxicity absence

At renewal, document continued indication, absence of unacceptable toxicity (e.g., hypersensitivity including anaphylaxis), and evidence of clinical benefit (or justification for dose escalation). Clinical benefit may be shown by improvements in growth, LDL, HDL, triglycerides, or AST/ALT; if escalating dose, provide rationale tied to suboptimal response criteria (poor growth, deteriorating biochemical markers, or lipid parameters).

Clinical Evidence

Phase 3Key trial: Phase 3 trial of sebelipase alfa in LAL deficiency (NEJM 2015)

PI Jul 2025Primary reference: Kanuma package insert, Alexion Pharmaceuticals, July 2025

Dose optionsDosing summary: 1 mg/kg q2w (pedi/adu), escalate to 3 mg/kg q2w; infants: 1 mg/kg qw with escalation to 3 mg/kg or 5 mg/kg if suboptimal

Key evidence sources include the Kanuma package insert (Alexion Pharmaceuticals, July 2025) and the Phase 3 trial of sebelipase alfa in LAL deficiency (Burton et al., NEJM 2015). These references support the efficacy of sebelipase alfa in patients with LAL deficiency and form the basis for the dosing, eligibility, and renewal criteria in this policy.

Background & Definitions

Background: Kanuma (sebelipase alfa) is an FDA-approved enzyme replacement therapy for Lysosomal Acid Lipase deficiency and is designated an orphan drug. The policy describes eligibility requirements, recommended dosing regimens (including escalation pathways for suboptimal response), and renewal requirements tied to clinical markers of benefit to guide appropriate use and authorization.

Term	Definition
LAL deficiency	Lysosomal Acid Lipase deficiency diagnosed by biallelic pathogenic LIPA variants or deficient LAL enzyme activity.