CurrentCignaPolicy IP0163

Gaucher Disease - Enzyme Replacement Therapy - Elelyso

Defines prior authorization requirements, clinical coverage criteria, dosing limits, and coding for Elelyso for treatment of Gaucher disease for Cigna-administered health benefit plans.

Policy Summary

PayerCigna

PolicyGaucher Disease - Enzyme Replacement Therapy - Elelyso

Policy CodePolicy IP0163

Change TypeRevised clinical criteria and added supportive-use criteria

Effective Date

Next Review DateJun 15, 2025

Key ActionObtain prior authorization; approvals are provided for 1 year when coverage criteria for Type 1 or Type 3 are met.

SourceLink

POLICY UPDATE CHANGES

Policy name changed from 'Taliglucerase' to 'Gaucher Disease - Enzyme Replacement Therapy - Elelyso.'

Gaucher Disease - Type 1: added qualifier 'Type 1' and note that Type 1 is non-neuronopathic; added age requirement of ≥ 4 years and dosing information; removed prior statement allowing Type 3 with at least one systemic manifestation.

Gaucher Disease - Type 3 was added as an 'Other Use with Supportive Evidence' with approval criteria and a dosing cap.

>=4Min age for approval

60 U/kgMax dose Type 1

120 U/kgMax dose Type 3

J3060Covered code

Policy Summary

PayerCigna

PolicyGaucher Disease - Enzyme Replacement Therapy - Elelyso

Policy CodePolicy IP0163

Change TypeRevised clinical criteria and added supportive-use criteria

Effective Date

Next Review DateJun 15, 2025

Key ActionObtain prior authorization; approvals are provided for 1 year when coverage criteria for Type 1 or Type 3 are met.

SourceLink

On This Page

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Coverage and Medical Necessity Criteria

Initial Therapy (FDA-Approved Indication)

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

Type 1 approvals

A) Patient is >= 4 years of age.
B) Diagnosis established by ONE of:
- i) Demonstration of deficient beta-glucocerebrosidase activity in leukocytes or fibroblasts.
- ii) Molecular genetic testing documenting biallelic pathogenic variants in the glucocerebrosidase (GBA) gene.
C) Elelyso 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.

Other Uses with Supportive Evidence

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

Type 3 approvals

A) Patient is >= 4 years of age.
B) Diagnosis established by ONE of:
- i) Demonstration of deficient beta-glucocerebrosidase activity in leukocytes or fibroblasts.
- ii) Molecular genetic testing documenting biallelic pathogenic variants in the glucocerebrosidase (GBA) gene.
C) Clinical use restrictions
- i) Medication is not being used for the management of neurological manifestations (examples: abnormal ocular movement, auditory impairment, cognitive impairment, seizures).

Elelyso (taliglucerase alfa) for any use other than the approved Type 1 indication and the specified Type 3 supportive use is not medically necessary. The policy explicitly states that Elelyso for any other use is considered not medically necessary and that the list of such uses may not be all-inclusive; criteria will be updated as new published data become available.

Concomitant use of Elelyso with other therapies approved for Gaucher disease has not been evaluated and therefore is not supported by the available evidence. Examples of other approved Gaucher therapies include Cerdelga (eliglustat), Cerezyme (imiglucerase), VPRIV (velaglucerase alfa), and Zavesca (miglustat).

Approvals are limited in scope. For Type 3 (chronic neuronopathic) Gaucher disease, coverage is restricted to management of non‑neurological, systemic manifestations — specifically when the medication is not being used to manage neurological manifestations and is being used to manage impaired growth, hematologic, or visceral symptoms (examples: splenomegaly, hepatomegaly, anemia, thrombocytopenia). Neurologic features such as abnormal ocular movement, auditory impairment, cognitive impairment, and seizures are examples of manifestations that would not be treated under the Type 3 supportive use criteria.

Elelyso must be prescribed by or in consultation with an appropriate specialist (geneticist, endocrinologist, metabolic disorder subspecialist, or a physician who specializes in lysosomal storage disorders). Dosing limits apply to each approved indication and must be followed (see dosing thresholds in the coding/thresholds section); receipt of sample product does not meet coverage requirements.

Coding and Dosing Limits

Considered Medically Necessary when criteria metHCPCSCovered

J3060

Injection, taliglucerase alfa, 10 units

Per-dose maximum — dosing limits

Type 1 (per-dose maximum)Each individual dose must not exceed 60 U/kg administered intravenously, no more frequently than once every 2 weeks.

Type 3 (per-dose maximum)Each individual dose must not exceed 120 U/kg administered intravenously, no more frequently than once every 2 weeks.

Frequency constraintFor both Type 1 and Type 3, doses are given IV no more frequently than once every 2 weeks.

Age-related notePolicy specifies patient age ≥ 4 years for approval of Elelyso for both Type 1 and Type 3.

Prescriber, Prior Authorization, and Documentation Requirements

Prior Authorization

Prior authorization required (1-year approvals)

Prior authorization is required for Elelyso. Approvals are provided for 1 year when the coverage criteria are met.

Prior authorization required for prescription benefit coverage of Elelyso.
Approval duration: 1 year when criteria for Type 1 (or Type 3 under supportive evidence) are met.

Step Therapy

Concomitant therapy not evaluated

Concomitant use of Elelyso with other treatments approved for Gaucher disease has not been evaluated and is not supported by evidence.

Examples of other approved therapies include eliglustat (Cerdelga), imiglucerase (Cerezyme), velaglucerase alfa (Vpriv), and miglustat (Zavesca).
Concomitant use listed as not evaluated in the policy.

Diagnostic Definitions and Related Criteria

Diagnostic criteria for Gaucher disease

Primary diagnostic methodsDemonstration of deficient β-glucocerebrosidase activity in leukocytes or fibroblasts.

Genetic testingMolecular genetic testing documenting biallelic pathogenic variants in the glucocerebrosidase (GBA) gene.

Diagnostic standardDiagnosis is established by either enzyme assay (leukocytes or fibroblasts) or by identification of biallelic pathogenic GBA variants.

Background

Gaucher disease is an autosomal recessive lysosomal storage disorder caused by deficient β‑glucocerebrosidase activity leading to accumulation of glucosylceramide in visceral organs. Clinical presentations vary by subtype: Type 1 is non‑neuronopathic and typically presents with visceromegaly, cytopenias, and skeletal disease, while Type 3 is chronic neuronopathic and includes both systemic and neurologic manifestations.

Diagnosis is established by demonstration of deficient enzyme activity in leukocytes or fibroblasts or by molecular genetic testing documenting biallelic pathogenic variants in the GBA gene. Treatment with Elelyso is intended to address systemic manifestations of Gaucher disease through enzyme replacement to reduce substrate accumulation.