Intravenous Enzyme Replacement Therapy (Cerezyme, Elelyso, VPRIV) for Type 1 Gaucher Disease

Coverage Summary

UnitedHealthcare medical benefit drug policy for Intravenous Enzyme Replacement Therapy (ERT) for Type 1 Gaucher disease. This policy defines coverage criteria, initial and continuation authorization rules, dosing limits, preferred product designation, and the applicable HCPCS/J-codes and diagnosis code for intravenous ERTs (imiglucerase, taliglucerase, velaglucerase).

Coverage stance: Covered with criteria. ERT (Cerezyme, Elelyso, VPRIV) is covered when policy-specified criteria are met for initial and continuation therapy. Initial and continuation authorizations are each limited to no more than 12 months. VPRIV (velaglucerase) is identified as the preferred agent and use of Cerezyme (imiglucerase) or Elelyso (taliglucerase) requires meeting the policy's step criteria (failure of or hypersensitivity to VPRIV).

Applicable billing codes and unit definitions are specified (examples: J1786 for imiglucerase per 10 units, J3060 for taliglucerase per 10 units, J3385 for velaglucerase per 100 units) and the covered diagnosis code referenced is E75.22 (Gaucher Disease).

Initial Therapy Criteria

General - Enzyme replacement therapy considered medically necessary 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 (example manifestations: moderate to severe anemia, thrombocytopenia, bone disease, hepatomegaly, splenomegaly)
Dosing is in accordance with FDA approved labeling

Elelyso (taliglucerase) specific - Initial therapy

Elelyso is medically necessary 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

Cerezyme (imiglucerase) specific - Initial therapy

Cerezyme is medically necessary 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

Continuation Therapy Criteria

General - Continuation of therapy

Covered for continuation 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)
Dosing is in accordance with FDA approved labeling

Elelyso - Continuation of therapy

Covered for continuation 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)

Product-specific Constraints

VPRIV (velaglucerase) specific

VPRIV is the preferred enzyme replacement therapy.

Indicated for long-term ERT in pediatric and adult patients with Type 1 Gaucher disease

Follow FDA labeling for dosing

Cerezyme - Additional dosing limit

When Cerezyme is used, the following dosing constraint applies:

Dose does not exceed 60 units/kg every 2 weeks<= 60 units/kg q2w

Initial authorization will be for no more than 12 months

Applicable Codes

Drug HCPCS/J-codes (unit definitions)HCPCS

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

Diagnosis codes referencedICD-10

E75.22

Gaucher Disease

Provider Actions

Prior Authorization

Initial and continuation authorization required

Initial and continuation authorizations are required and each authorization (initial or continuation) is limited to no more than 12 months. Documentation must support the diagnosis of Type 1 Gaucher disease, presence of symptomatic disease for initial therapy, demonstration of positive clinical response for continuation, and dosing must follow FDA labeling.

J1786
J3060
J3385

Documentation Required

Documentation of clinical response

For continuation requests, submit documentation showing a positive clinical response such as reduced severity or resolution of anemia, thrombocytopenia, bone disease, hepatomegaly, or splenomegaly.

Clinical Evidence

+2.43 g/dLMean hemoglobin increase at 12 months (velaglucerase 60 U/kg)

+65.9%Mean platelet increase at 12 months (velaglucerase 60 U/kg)

-50.4%Mean spleen volume reduction at 12 months (velaglucerase 60 U/kg)

Cochrane review conclusion: during the first year of treatment, different recombinant glucocerebrosidases are bio-similar and non-inferior in safety and efficacy for surrogate biological response parameters; lower doses (30–45 U/kg every 2–4 weeks) were generally as effective as 60 U/kg for assessed clinical outcomes in the first year.

Background evidence: clinical studies and cohort data report that enzyme replacement therapy improves hematologic and visceral disease parameters — for example, velaglucerase produced mean increases in hemoglobin and platelet counts and marked reductions in spleen and liver volumes at 12 months — supporting ERT efficacy in Type 1 Gaucher disease.

Background

Gaucher disease is an autosomal recessive disorder caused by deficient glucocerebrosidase with accumulation of glucocerebroside in reticuloendothelial tissues. Type 1 is the most common, non-neuronopathic form and is characterized by systemic visceral and hematologic manifestations without primary central nervous system involvement.

Type 1 (non-neuronopathic) Gaucher disease is the primary indication for intravenous enzyme replacement therapies imiglucerase (Cerezyme), taliglucerase (Elelyso), and velaglucerase (VPRIV); these agents are indicated for long-term ERT in pediatric and adult patients with Type 1 Gaucher disease.

Clinical evidence supports that ERTs improve hematologic (hemoglobin, platelet counts) and visceral (spleen and liver volumes) parameters in Type 1 Gaucher disease, with randomized and cohort studies demonstrating clinically meaningful improvements within the first 12 months.

Definitions and Thresholds

Positive clinical responseReduced severity or resolution of anemia, thrombocytopenia, bone disease, hepatomegaly, or splenomegaly.

Initial authorizationAuthorization period for new therapy, limited to no more than 12 months per policy.

Cerezyme dosing limitDose does not exceed 60 units/kg every 2 weeks

Revision History

07/01/2025clarifiedLatest

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

06/01/2025revised

Supporting Information - Updated References section to reflect the most current information (06/01/2025). Archived previous policy version 2024D0048O.