CurrentColorado Rocky Mountain Health PlansPolicy 2025D0048P

Intravenous Enzyme Replacement Therapy (ERT) for Gaucher Disease

Clinical coverage criteria for intravenous enzyme replacement therapies (Cerezyme/imiglucerase, Elelyso/taliglucerase alfa, VPRIV/velaglucerase alfa) for treatment of Gaucher disease (Types 1 and 3) under UnitedHealthcare commercial Medical Benefit Drug Policy.

Policy Summary

PayerColorado Rocky Mountain Health Plans

PolicyIntravenous Enzyme Replacement Therapy (ERT) for Gaucher Disease

Policy CodePolicy 2025D0048P

Change TypeTemplate and reference updates

Effective DateJun 1, 2025

Next Review Date

Key ActionSubmit authorization requests with documentation of diagnosis, symptomatic disease, dosing consistent with FDA labeling, and for continuation include evidence of positive clinical response; authorizations are limited to up to 12 months.

SourceLink

POLICY UPDATE CHANGES

Template Update: Benefit Considerations updated on 07/01/2025; References updated 06/01/2025.

3intravenous ERT products listed

Type 1, Type 3Gaucher disease types with explicit coverage criteria

12 monthsmaximum initial and continuation authorization period

60 U/kgdose cap specified for Type 3 (VPRIV and Cerezyme conditions)

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Criteria for Enzyme Replacement Therapy (ERT)

General — ERT (Cerezyme, Elelyso, VPRIV) for Type 1 Gaucher disease

ERT is proven/medically necessary for Type 1 Gaucher disease 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 authorization will be for no more than 12 months
Continuation authorizations handled separately

Continuation therapy — Type 1 Gaucher disease (all products)

For continuation of therapy ALL of the following are required:

ALL of the following

Documentation of positive clinical response to therapy (e.g., reduced severity or resolution of anemia, thrombocytopenia, bone disease, hepatomegaly, splenomegaly)
Dosing is in accordance with FDA-approved labeling
Continuation authorization will be for no more than 12 months

Elelyso (taliglucerase alfa) — initial therapy (Community Plan specific)

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 the following
- 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

Cerezyme (imiglucerase) — initial therapy (Community Plan specific)

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 the following
- 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 alfa) — Type 3 Gaucher disease

VPRIV is medically necessary for Type 3 Gaucher disease 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 (imiglucerase) — Type 3 Gaucher disease

Cerezyme is medically necessary for Type 3 Gaucher disease 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 the following
- 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

Relevant Billing and Diagnosis Codes

Drug HCPCS codes (reference)HCPCS

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

Diagnosis codes (reference)ICD-10

E75.22

Gaucher disease

Authorization, Documentation, and Prior Authorization Requirements

Prior Authorization

Authorization required with clinical documentation

Initial and continuation authorizations require documentation of a diagnosis of Gaucher disease with symptomatic disease, dosing that is consistent with FDA‑approved labeling, and for continuation documentation of a positive clinical response. Initial and continuation authorizations are limited to no more than 12 months per request.

Affected codes: J1786, J3060, J3385, E75.22

Documentation Required

Failure or hypersensitivity to preferred agent

When requesting Cerezyme or Elelyso after prior VPRIV therapy, provide documentation of either failure to meet clinical goals on VPRIV (e.g., persistent anemia, thrombocytopenia, bone disease, hepatomegaly, or splenomegaly) or documented hypersensitivity to VPRIV.

Affected codes: J3385, J1786, J3060

Clinical Context

Gaucher disease is an inherited autosomal recessive disorder caused by deficient glucocerebrosidase, leading to accumulation of glucocerebroside in reticuloendothelial tissues such as the liver, spleen, and bone marrow. Clinical subtypes include Type 1 (non‑neuronopathic, accounting for >90% of cases), Type 2 (acute neuronopathic with severe early neurologic involvement and poor prognosis), and Type 3 (chronic neuronopathic with systemic manifestations).

Intravenous enzyme replacement therapy (ERT) with recombinant glucocerebrosidases (imiglucerase/Cerezyme, taliglucerase/Elelyso, velaglucerase/VPRIV) is indicated principally to treat the visceral and hematologic manifestations of Gaucher disease — for example, anemia, thrombocytopenia, hepatomegaly, splenomegaly, and bone disease — and has been shown to improve these systemic features.

ERT is not effective for primary central nervous system (CNS) involvement; neurologic manifestations of neuronopathic Gaucher disease (Types 2 and 3) do not respond to ERT, so treatment decisions for patients at risk of neuronopathic disease should be guided by non‑neurologic manifestations rather than expectation of neurologic benefit.

Defined Terms

Definitions

Symptomatic diseaseExamples include moderate to severe anemia, thrombocytopenia, bone disease, hepatomegaly, or splenomegaly.

Positive clinical responseReduction in severity or resolution of anemia, thrombocytopenia, bone disease, hepatomegaly, splenomegaly as documented by clinical assessment and labs/imaging.

Recent Policy Changes

2025-07-01clarifiedLatest

Template update: Benefit Considerations section updated; administrative/template changes only with no substantive clinical criteria changes.

2025-06-01clarified

References section updated to reflect the most current information and previous policy version 2024D0048O archived.

Quick Policy Metrics

Policy thresholds

Authorization durationInitial and continuation authorization up to 12 months

Dose cap for Type 3 (VPRIV and Cerezyme when specified)<= 60 units/kg every 2 weeks

Intravenous Enzyme Replacement Therapy (ERT) for Gaucher Disease

Criteria for Enzyme Replacement Therapy (ERT)

Relevant Billing and Diagnosis Codes

Authorization, Documentation, and Prior Authorization Requirements

Clinical Context

Defined Terms

Recent Policy Changes

Other Policies to Review

Quick Policy Metrics