CurrentCareSourcePolicy N/A

Vpriv (velaglucerase) — Enzyme replacement therapy for Gaucher disease (Type 1, Type 3)

Defines prior authorization, coverage criteria, dosing limits, and specialist prescriber requirements for Vpriv (velaglucerase) for Type 1 and Type 3 Gaucher disease; applies to CareSource medical benefit coverage decisions.

Policy Summary

PayerCareSource

PolicyVpriv (velaglucerase) — Enzyme replacement therapy for Gaucher disease (Type 1, Type 3)

Policy CodePolicy N/A

Change TypeMaterial updates — added Type 3 coveragerevised Type 1 age and genetic testing requirementsclarifying exclusions

Effective Date

Next Review DateApr 2, 2025

Key ActionObtain prior authorization and document diagnosis with enzyme testing or molecular genetic testing showing biallelic pathogenic GBA variants; ensure prescribing by or in consultation with an appropriate specialist.

SourceLink

POLICY UPDATE CHANGES

Gaucher Disease - Type 3 was added as an Other Use with Supportive Evidence with specific coverage criteria and dosing.

For Type 1, age > 4 years was added and genetic testing requirement specified as biallelic pathogenic variants in GBA.

Concomitant use with other approved Gaucher disease therapies is listed as not recommended for approval.

2Covered indications

>4Min age

60 U/kgMax dose (Type 1)

120 U/kg

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Coverage Criteria for Vpriv (velaglucerase)

FDA-Approved Indication: Gaucher Disease - Type 1

Approve for 1 year if the patient meets ALL of the following (A, B, and C):

Type 1 approval criteria: A) Patient is > 4 years of age;

Type 1 diagnosis (one of): B) Diagnosis established by ONE of: i. Demonstration of deficient β-glucocerebrosidase activity in leukocytes or fibroblasts; OR ii. Molecular genetic testing documenting biallelic pathogenic variants in the glucocerebrosidase (GBA) gene.

Prescriber requirement: C) Vpriv is prescribed by or in consultation with a geneticist, endocrinologist, a metabolic disorder sub-specialist, or a physician who specializes in the treatment of lysosomal storage disorders.

Dosing: Each individual dose must not exceed 60 U/kg administered intravenously no more frequently than once every 2 weeks.

Other Uses with Supportive Evidence: Gaucher Disease - Type 3

Approve for 1 year if the patient meets ALL of the following (A, B, C, and D):

Type 3 approval criteria: A) Patient is > 4 years of age;

Type 3 diagnosis (one of): B) Diagnosis established by ONE of: i. Demonstration of deficient β-glucocerebrosidase activity in leukocytes or fibroblasts; OR ii. Molecular genetic testing documenting biallelic pathogenic variants in the glucocerebrosidase (GBA) gene.

Clinical use limitations (both required): C) The patient meets BOTH of the following: i. Medication is not being used for the management of neurological manifestations (examples may include abnormal ocular movement, auditory impairment, cognitive impairment, and seizures); AND ii. Medication is being used for the management of impaired growth, hematologic, or visceral symptoms (examples of visceral symptoms include splenomegaly and hepatomegaly; examples of hematologic symptoms include anemia and thrombocytopenia).

Prescriber requirement: D) Vpriv is prescribed by or in consultation with a geneticist, endocrinologist, a metabolic disorder sub-specialist, or a physician who specializes in the treatment of lysosomal storage disorders.

Dosing: Each individual dose must not exceed 120 U/kg administered intravenously no more frequently than once every 2 weeks.

Concomitant use of Vpriv with other therapies approved for Gaucher disease is not recommended for approval. The policy notes that concomitant use has not been evaluated and provides examples of other approved Gaucher treatments, including eliglustat (Cerdelga), taliglucerase alfa (Elelyso), imiglucerase (Cerezyme), and miglustat (Zavesca).

Coverage of Vpriv is not recommended for any circumstances that are not explicitly listed in the Recommended Authorization Criteria. The policy states that criteria will be updated as new published data become available and that requests outside the listed criteria should be considered non-recommended for approval.

Requests to use Vpriv concurrently with other therapies approved for Gaucher disease are considered not recommended and would not be approvable under this policy. The document emphasizes that concomitant use has not been evaluated and lists representative approved agents as examples.

Diagnostic Evidence Acceptable for Coverage

Molecular genetic testing documenting biallelic pathogenic variants in the GBA gene is acceptable diagnostic evidence equivalent to enzyme activity testing

Molecular genetic testing documenting biallelic pathogenic variants in the GBA gene is acceptable diagnostic evidence equivalent to enzyme activity testing.

Acceptable diagnostic methods (one of): Molecular genetic testing documenting biallelic pathogenic variants in the glucocerebrosidase (GBA) gene; OR demonstration of deficient β-glucocerebrosidase activity in leukocytes or fibroblasts.

When molecular genetic testing is used to establish diagnosis, documentation of biallelic pathogenic variants in GBA is required.

Product and Dosing Limits

Productmixed

Vpriv

velaglucerase intravenous infusion - product name (no explicit HCPCS/CPT provided in document)

inv-07: Type 1 per-dose limit — <= 60 U/kg every ≥2 weeks

Per-dose maximumEach individual dose must not exceed 60 U/kg administered intravenously

Dosing frequencyNo more frequently than once every 2 weeks (≥2 weeks between doses)

Approval duration linkDosing limit applies to FDA‑approved Type 1 indication with 1‑year authorization when criteria met

inv-08: Type 3 per-dose limit — <= 120 U/kg every ≥2 weeks

Per-dose maximumEach individual dose must not exceed 120 U/kg administered intravenously

Dosing frequencyNo more frequently than once every 2 weeks (≥2 weeks between doses)

Prior Authorization and Documentation Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is recommended for medical benefit coverage of Vpriv. Approval is for 1 year when the patient meets the policy Criteria and Dosing for the listed indications. Requests for doses outside the established dosing documented in this policy will be considered on a case‑by‑case basis by a clinician (Medical Director or Pharmacist). Because of the specialized skills required for evaluation, diagnosis, and monitoring of patients treated with Vpriv, the medication must be prescribed by or in consultation with an appropriate specialist (geneticist, endocrinologist, metabolic disorder subspecialist, or equivalent).

Approval duration: 1 year when criteria are met
Dosing limits apply per indication (see policy dosing sections)

Note

Dose Exceptions

Requests for doses outside established dosing will be reviewed on a case‑by‑case basis by a clinician (e.g., Medical Director or Pharmacist).

Member Eligibility

Approval and continuation of Vpriv require that the patient meet the policy's specified eligibility criteria for the indicated Gaucher disease type. For Type 1 and Type 3 indications, authorization is for 1 year when all criteria are met, including minimum age (>4 years), diagnostic documentation (enzyme activity testing or molecular genetic testing showing biallelic pathogenic GBA variants), and that the drug is prescribed by or in consultation with an appropriate specialist (geneticist, endocrinologist, metabolic disorder sub-specialist, or physician who specializes in lysosomal storage disorders). Dosing limits must also be followed (Type 1: ≤ 60 U/kg IV no more frequently than once every 2 weeks; Type 3: ≤ 120 U/kg IV no more frequently than once every 2 weeks).

Disease Definitions

inv-14: Type 1 Gaucher disease — non-neuronopathic variant presenting with splenomegaly, hepatomegaly, cytopenias, and skeletal complications

DefinitionType 1 Gaucher disease (non‑neuronopathic): presents with splenomegaly, hepatomegaly, cytopenias (anemia, thrombocytopenia), and skeletal complications

Typical prevalenceType 1 is the most prevalent phenotype, accounting for the majority of cases

Diagnostic methodsDiagnosis established by demonstration of deficient β‑glucocerebrosidase activity in leukocytes or fibroblasts or by molecular genetic testing showing biallelic pathogenic GBA variants

inv-15: Type 3 Gaucher disease — chronic neuronopathic form with neurologic, hematologic and visceral symptoms; ERT ameliorates systemic but not neurologic disease

DefinitionType 3 Gaucher disease (chronic neuronopathic): chronic neuronopathic form with neurologic, hematologic, and visceral symptoms

Background on Gaucher Disease and Therapy

Gaucher disease is an autosomal recessive lysosomal storage disorder caused by deficient β‑glucocerebrosidase, leading to accumulation of glucosylceramide in visceral organs and bone marrow. Type 1 is the non‑neuronopathic form, typically presenting with splenomegaly, hepatomegaly, cytopenias, and skeletal complications. Type 3 is a chronic neuronopathic form characterized by neurologic, hematologic, and visceral manifestations; enzyme replacement therapy can ameliorate systemic (hematologic and visceral) disease but does not address neurological involvement. Diagnosis may be established by demonstration of deficient enzyme activity in leukocytes or fibroblasts or by molecular genetic testing documenting biallelic pathogenic variants in the GBA gene.

Policy Revision History

2025-04-02annual_reviewLatest

Annual revision completed; no criteria changes noted during this review.

2024-07-17selected_revision

Selected revision recorded (details in prior entry); no criteria changes at this review.

2024-04-10annual_review