CurrentUnitedHealthcarePolicy 2025D0048P

Intravenous Enzyme Replacement Therapy (ERT) for Gaucher Disease

Defines UnitedHealthcare coverage criteria for intravenous ERT agents (Cerezyme/imiglucerase, Elelyso/taliglucerase, VPRIV/velaglucerase) for treatment of Type 1 and Type 3 Gaucher disease, including initial and continuation authorization criteria, dosing limits, preferred product, and applicable HCPCS/J-codes and ICD-10 diagnosis code.

Policy Summary

PayerUnitedHealthcare

PolicyIntravenous Enzyme Replacement Therapy (ERT) for Gaucher Disease

Policy CodePolicy 2025D0048P

Change TypeTemplate update; References updated

Effective DateJun 1, 2025

Next Review Date

Key ActionInitial and continuation authorizations are limited to no more than 12 months and continuation requires documentation of positive clinical response.

POLICY UPDATE CHANGES

07/01/2025 Template Update - Updated Benefit Considerations.

06/01/2025 Supporting Information - Updated References; Archived previous policy version 2024D0048O.

3Products referenced

Type 1 & Type 3Covered Indications

<=60 U/kg q2wkVPRIV dosing ceiling (Type 3)

Coverage Summary

Scope: This policy defines UnitedHealthcare coverage criteria for intravenous enzyme replacement therapies — Cerezyme (imiglucerase), Elelyso (taliglucerase), and VPRIV (velaglucerase) — for the treatment of Type 1 and Type 3 Gaucher disease. Coverage stance: these products are covered with criteria, with VPRIV designated as the preferred enzyme replacement therapy. Authorization limits: initial and continuation authorizations are each limited to no more than 12 months, and dosing must follow FDA labeling and applicable J-codes (e.g., J1786, J3060, J3385).

General - Cerezyme, Elelyso, VPRIV for Type 1 Gaucher disease

Covered when ALL of the following are met:

ALL of the following

Diagnosis of Type 1 Gaucher disease
Symptomatic disease (e.g., moderate to severe anemia, thrombocytopenia, bone disease, hepatomegaly, splenomegaly)
Dosing is in accordance with FDA approved labeling
Initial and continuation dosing must match FDA labeling
Initial authorization will be for no more than 12 months

Product-specific Initial Therapy Criteria

Elelyso (taliglucerase) specific initial therapy

Elelyso is medically necessary for Type 1 when ALL of the following are met:

ALL of the following

Diagnosis of Type 1 Gaucher disease
ONE of
- History of failure of VPRIV due to failure to meet clinical goals (e.g., persistent anemia, thrombocytopenia, bone disease, hepatomegaly, or splenomegaly) despite VPRIV therapy
- History of failure of VPRIV due to hypersensitivity to VPRIV therapy
Dosing is in accordance with FDA approved labeling
Initial authorization will be for no more than 12 months

Cerezyme (imiglucerase) specific initial therapy for Type 1

Cerezyme is medically necessary for Type 1 when ALL of the following are met:

ALL of the following

Diagnosis of Type 1 Gaucher disease
ONE of
- History of failure of VPRIV due to failure to meet clinical goals despite VPRIV therapy
- History of failure of VPRIV due to hypersensitivity to VPRIV therapy
Dosing is in accordance with FDA approved labeling
Initial authorization will be for no more than 12 months

VPRIV (velaglucerase) for Type 3 Gaucher disease - initial

VPRIV is medically necessary for Type 3 when ALL of the following are met:

ALL of the following

Diagnosis of Type 3 Gaucher disease
Symptomatic disease (e.g., moderate to severe anemia, thrombocytopenia, bone disease, hepatomegaly, splenomegaly)
Dose does not exceed 60 units/kg every 2 weeks<= 60 units/kg every 2 weeks
Initial authorization will be for no more than 12 months

Cerezyme for Type 3 Gaucher disease - initial

Cerezyme is medically necessary for Type 3 when ALL of the following are met:

ALL of the following

Diagnosis of Type 3 Gaucher disease
Symptomatic disease (e.g., moderate to severe anemia, thrombocytopenia, bone disease, hepatomegaly, splenomegaly)
ONE of
- History of failure of VPRIV due to failure to meet clinical goals despite VPRIV therapy
- History of failure of VPRIV due to hypersensitivity to VPRIV therapy
Dose does not exceed 60 units/kg every 2 weeks

Continuation Therapy Criteria

VPRIV (velaglucerase) for Type 3 Gaucher disease - continuation

Covered when ALL of the following are met:

ALL of the following

Documentation of positive clinical response to therapy (e.g., reduced severity or resolution of anemia, thrombocytopenia, bone disease, hepatomegaly, splenomegaly)
examples listed in policy
Dose does not exceed 60 units/kg every 2 weeks<= 60 units/kg every 2 weeks
Continuation authorization will be for no more than 12 months

Cerezyme for Type 3 Gaucher disease - continuation

Covered when ALL of the following are met:

ALL of the following

Documentation of positive clinical response to therapy
Dose does not exceed 60 units/kg every 2 weeks<= 60 units/kg every 2 weeks
Continuation authorization will be for no more than 12 months

Provider Actions

Prior Authorization

Authorization duration limits

Initial and continuation authorizations are each limited to no more than 12 months. Ensure authorization requests specify the requested authorization period (initial or continuation) consistent with policy limits.

Documentation Required

Document clinical response for continuation

For continuation requests, submit documentation demonstrating a positive clinical response to therapy. Acceptable evidence includes reduced severity or resolution of disease manifestations such as anemia, thrombocytopenia, bone disease, hepatomegaly, or splenomegaly.

Examples of clinical response: improved hemoglobin
Increased platelet count or reduced bleeding
Reduction in spleen or liver volume; improvement in bone disease

Billing Rule

Follow FDA approved dosing and J-code usage

Dosing must follow FDA‑approved labeling for the selected product. Bill the appropriate HCPCS J‑code and units for the product administered (examples: J1786 for imiglucerase, J3060 for taliglucerase alfa, J3385 for velaglucerase alfa) using the product‑specific unit definitions.

J1786 — Injection, imiglucerase, 10 units
J3060 — Injection, taliglucerase alfa, 10 units
J3385 — Injection, velaglucerase alfa, 100 units

Prior Authorization

Product selection preference

VPRIV (velaglucerase) is designated as the preferred enzyme replacement therapy. Use of non‑preferred products (Cerezyme, Elelyso) may require documentation of prior failure to, or hypersensitivity with, VPRIV before approval.

Non‑preferred product approval may require history of VPRIV failure to meet clinical goals (persistent anemia, thrombocytopenia, bone disease, hepatomegaly, or splenomegaly)
Or documentation of hypersensitivity to VPRIV

Clinical Evidence

3Products referenced (Cerezyme, Elelyso, VPRIV)

Type 1 & Type 3Covered indications by policy

<=60 U/kg q2wkVPRIV dosing ceiling for Type 3 and continuation

Evidence: A 2015 Cochrane review found that during the first year of treatment different recombinant glucocerebrosidases are bio‑similar and non‑inferior in safety and efficacy for surrogate biological response parameters. In a multinational phase 3 velaglucerase alfa trial (60 U/kg every other week), mean hemoglobin increased by +2.43 g/dL at 12 months and mean spleen volume decreased by -50.4% in the 60 U/kg arm; platelet counts also showed clinically meaningful increases.

Background

Background: Gaucher disease is an autosomal recessive deficiency of glucocerebrosidase causing glucocerebroside accumulation in liver, spleen, bone marrow and other tissues. Type 1 is the most common (>90%) and is non‑neuronopathic with systemic manifestations; Type 2 is acute neuronopathic with severe early neurologic disease and early death; Type 3 is chronic neuronopathic with subacute neurologic symptoms plus systemic disease. For informational purposes, FDA labeling indicates Cerezyme, Elelyso, and VPRIV are approved for Type 1 (with age indications varying by product), and ERT generally reverses visceral manifestations but does not improve primary central nervous system involvement in Types 2 and 3. Differential responses: neurologic manifestations of neuronopathic disease do not appear to respond to ERT, though visceral disease in Type 3 can respond to therapy.

Definitions: Symptomatic disease — examples include moderate to severe anemia, thrombocytopenia, bone disease, hepatomegaly, splenomegaly.

Policy Summary

PayerUnitedHealthcare

PolicyIntravenous Enzyme Replacement Therapy (ERT) for Gaucher Disease

Policy CodePolicy 2025D0048P

Change TypeTemplate update; References updated

Effective DateJun 1, 2025

Next Review Date

Key ActionInitial and continuation authorizations are limited to no more than 12 months and continuation requires documentation of positive clinical response.

On This Page

OpenPayer

Intravenous Enzyme Replacement Therapy (ERT) for Gaucher Disease

Coverage Summary

Product-specific Initial Therapy Criteria

Continuation Therapy Criteria

Provider Actions

Applicable Codes

Clinical Evidence

Background

Revision History

J1786	Injection, imiglucerase, 10 units
J3060	Injection, taliglucerase alfa, 10 units
J3385	Injection, velaglucerase alfa, 100 units