CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Lumizyme (alglucosidase alfa) — Coverage Criteria

Policy governing coverage and authorization criteria for Lumizyme (alglucosidase alfa) for Neighborhood Health Plan of Rhode Island members, distinguishing Pharmacy benefit for Medicaid and Medical benefit for Commercial and MMP members.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyLumizyme (alglucosidase alfa) — Coverage Criteria

Policy CodePolicy N/A

Change TypeNo material changes

Effective Date04/01/2022

Next Review DateN/A

Key ActionObtain prior authorization; initial approvals are for 6 months and may be renewed for additional 6-month periods.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

6 monthsLength of initial authorization

20 mg/kg q2wDosing

J0221HCPCS

E74.02Covered diagnosis

46 vials/14dQuantity limit

Medicaid

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Coverage Criteria

Initial Therapy

Covered when ALL of the following are met:

Initial approval: Diagnosis of Pompe disease confirmed by deficiency of GAA enzyme activity OR detection of biallelic pathogenic GAA variants; AND documented baseline age-appropriate values (infantile-onset: muscle weakness, motor function, respiratory function, cardiac involvement, percent predicted FVC, and 6-MWT OR late-onset: FVC and 6-MWT); AND patient is not receiving other enzyme replacement therapies concurrently (e.g., avalglucosidase-alfa, cipaglucosidase alfa-atga); AND patient has no history of severe hypersensitivity including anaphylaxis to alglucosidase alfa; AND patient is not susceptible to fluid volume overload and does not have an acute underlying respiratory illness or compromised cardiac or respiratory function for whom fluid restriction is indicated.

MMP members who received this medication within the past 365 days are not subject to Step Therapy Requirements.

Continuation/Renewal

Authorization may be renewed when ALL of the following are met:

Renewal: Patient continues to meet universal and indication-specific criteria from initial approval; AND absence of unacceptable toxicity to the drug (examples include anaphylaxis, severe immune-mediated reactions, acute cardiorespiratory failure, cardiac arrhythmia during general anesthesia, etc.); AND patient is being monitored for antibody formation (including neutralizing antibodies); AND patient demonstrates stabilization or improvement compared to pretreatment age-appropriate baseline in relevant measures (infantile-onset: muscle strength, motor function, respiratory function, cardiac involvement, FVC, and 6-MWT OR late-onset: FVC and 6-MWT).

Use of Lumizyme in combination with other enzyme replacement therapies is not permitted. Specifically, concurrent administration with agents such as avalglucosidase-alfa (Nexviazyme) or cipaglucosidase alfa-atga (Pombiliti) is excluded from coverage.

For members enrolled in Medicare plans, these coverage criteria apply only in the absence of a National Coverage Determination (NCD) or Local Coverage Determination (LCD). When an applicable NCD or LCD exists, that determination takes precedence; Neighborhood will consider individual requests in light of the NCD/LCD and information provided by the prescriber.

Patients with a history of severe hypersensitivity, including anaphylaxis, to alglucosidase alfa (Lumizyme) should not receive this therapy. A prior severe hypersensitivity reaction to the product is a contraindication to treatment.

Coding and Billing

HCPCS CodesHCPCSCovered

J0221

Injection, alglucosidase alfa, (Lumizyme), 10 mg

NDCNDC

58468-0160-xx

Lumizyme 50 mg single-dose vial for injection

Covered Diagnosis CodesICD-10Covered

E74.02

Pompe disease

inv-09: Quantity limit — Lumizyme 50 mg vial: max 46 vials every 14 days; equivalent to 230 billable units every 14 days

ProductLumizyme 50 mg vial

Quantity limitMaximum 46 vials every 14 days

Equivalent unitsNDC unit basis

Provider Actions and Requirements

Prior Authorization

Authorization duration and renewal

Coverage will be provided for 6 months and may be renewed for an additional 6-month period. Prior authorization is required per plan rules.

Initial authorization: 6 months
Renewals: additional 6-month periods

Prior Authorization

Prior authorization requirement

Prior authorization is required per plan criteria. Neighborhood will give individual consideration to requests based on the information submitted by the prescriber and other information available to the plan. Coverage criteria apply for Medicaid pharmacy and specified medical scopes and consider NCD/LCD where applicable; for Medicare-Medicaid Plan (MMP) members these criteria apply only in the absence of applicable NCD/LCD.

Prior authorization required for Pharmacy (Medicaid) and Medical (Commercial, MMP) scopes
MMP: apply plan criteria only when no NCD/LCD exists
Requests reviewed on individual clinical submission

Background

Pompe disease is an inherited deficiency of acid alpha-glucosidase (GAA) that leads to glycogen accumulation in muscle and other tissues; clinical manifestations vary from infantile-onset cardiomyopathy and profound muscle weakness to later-onset progressive myopathy and respiratory compromise. Enzyme replacement therapy with recombinant human GAA aims to replace the deficient enzyme, reduce glycogen storage, and improve motor and respiratory function.

Lumizyme (alglucosidase alfa) is a recombinant human GAA indicated for treatment of Pompe disease and is administered intravenously. Use of Lumizyme has been shown to improve respiratory and motor outcomes and survival in affected patients. Treatment requires confirmation of diagnosis (by decreased GAA enzyme activity or biallelic pathogenic GAA variants) and baseline documentation of age-appropriate clinical measures (for example, FVC, 6MWT, and muscle/respiratory/cardiac assessments) to permit monitoring of response and safety. Therapy should not be used in combination with other ERTs, and it is contraindicated in patients with prior severe hypersensitivity to alglucosidase alfa. For Medicare members, coverage is subject to NCD/LCD determinations when present.

Definitions

inv-19: Pompe disease definition — GAA deficiency diagnostic criteria (enzyme activity or biallelic pathogenic variants).

ConditionPompe disease (acid alpha-glucosidase [GAA] deficiency)

Diagnostic criteria 1Deficiency of GAA enzyme activity

Diagnostic criteria 2Detection of biallelic pathogenic variants in the GAA gene by molecular genetic testing

Initial Therapy Criteria

Initial Therapy — Initial dosing and baseline requirements

Initial dosing and baseline requirements:

Dose and baseline requirements: Dose: 20 mg/kg administered IV every 2 weeks; AND documentation of baseline age-appropriate clinical measures appropriate to disease onset (infantile: muscle weakness, motor, respiratory, cardiac, percent predicted FVC, 6-MWT; late-onset: FVC and 6-MWT); AND diagnosis confirmed by GAA enzyme activity deficiency or biallelic pathogenic GAA variants by molecular testing; AND not to be used in combination with other enzyme replacement therapies.

Authorization period: initial approval provided for 6 months per plan rules.

Continuation / Renewal Criteria

Renewal Criteria

Renewal requires clinical benefit and safety monitoring:

Renewal monitoring and benefit: No unacceptable toxicity from therapy (examples: anaphylaxis, immune-mediated cutaneous or systemic reactions, acute cardiorespiratory events); AND active monitoring for antibody formation including neutralizing antibodies; AND evidence of stabilization or improvement versus pretreatment baseline in specified measures (infantile-onset: muscle, motor, respiratory, cardiac, FVC, 6-MWT; late-onset: FVC and 6-MWT); AND continued fulfillment of universal and indication-specific criteria.

Renewals are provided at 6-month intervals.

Step Therapy

Step therapy condition	Applies to
MMP members who have previously received Lumizyme within the prior 365 days are exempt from step therapy requirements.
All other members: standard step therapy requirements apply per plan rules (see step therapy policy).

Quantity Limits

inv-23: Lumizyme 50 mg vial — 46 vials every 14 days

ProductLumizyme 50 mg vial

Limit46 vials every 14 days

Limit periodPer 14-day period

inv-24: Lumizyme (billing units) — 230 billable units every 14 days

Billing unit definition10 mg billable unit (HCPCS basis)

Max billable units230 billable units every 14 days

Equivalent vial calculation230 units corresponds to 46 vials of 50 mg per 14 days

Site of Care and Benefit Scope

Billing Rule

Site‑of‑care billing: Pharmacy vs Medical benefit

For Medicaid members the medication is available under the Pharmacy benefit; for Commercial and MMP members the medication is covered under the Medical benefit (infusion center or medical office).