CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Cerdelga (eliglustat) — Coverage for Gaucher Disease Type 1 (CYP2D6–guided)

Covers use of Cerdelga (eliglustat) for long-term treatment of adult patients with Gaucher disease type 1 when FDA-cleared CYP2D6 testing and the policy's approval criteria are met; applies to members of Neighborhood Health Plan of Rhode Island.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyCerdelga (eliglustat) — Coverage for Gaucher Disease Type 1 (CYP2D6–guided)

Policy CodePolicy N/A

Change TypeNo material change

Effective DateNov 15, 2020

Next Review DateFeb 1, 2025

Key ActionObtain prior authorization with documentation of Gaucher disease diagnosis and FDA-cleared CYP2D6 metabolizer status to qualify for up to 6 months of therapy.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

6 monthsauthorization period

CYP2D6required test

2 caps/daydose (EM/IM)

1 cap/daydose (PM)

Adult GD1indication

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Coverage Criteria for Cerdelga (eliglustat)

Initial Therapy

Covered when ALL of the following are met for initial approval:

Initial Approval Criteria: Patient is an adult with Gaucher disease type 1; Diagnosis confirmed by enzyme assay demonstrating a deficiency of beta-glucocerebrosidase (glucosidase) enzyme activity or by genetic testing; Documentation of CYP2D6 metabolizer status (extensive, intermediate, or poor) detected by an FDA-cleared test.

Authorization of 6 months may be granted.

Continuation Therapy

Continuation (re-authorization) covered when ALL of the following are met:

Continuation Criteria: Documentation that the patient continues to meet the criteria for initial approval; Documentation that the patient is not experiencing an inadequate response or any intolerable adverse events from therapy.

Authorization of 6 months may be granted.

Determination of CYP2D6 metabolizer status

Determination of CYP2D6 metabolizer status to guide eligibility and dosing for Cerdelga in adults with Gaucher disease type 1. Testing must be performed with an FDA‑cleared test.

CYP2D6 Testing Requirement: CYP2D6 metabolizer status (extensive, intermediate, or poor) must be determined using an FDA-cleared test to guide eligibility and dosing for Cerdelga in adults with Gaucher disease type 1.

Cerdelga is indicated only for adults with GD1 who are classified as EM, IM, or PM by an FDA-cleared test.

All indications for Cerdelga (eliglustat) other than the FDA‑approved indication for adult Gaucher disease type 1 (GD1) are considered experimental/investigational and not medically necessary. This policy limits coverage to the FDA‑approved population and does not authorize use for other diagnoses or off‑label indications.

Use of Cerdelga for non‑FDA‑approved indications (that is, any indication outside treatment of adults with Gaucher disease type 1 with documented CYP2D6 metabolizer status by an FDA‑cleared test) is not medically necessary and is not covered under this policy.

Covered Indications

Determination of CYP2D6 metabolizer status

Determination of CYP2D6 metabolizer status to guide eligibility and dosing for Cerdelga in adults with Gaucher disease type 1.

Covered Genetic Test Indication: Testing to determine CYP2D6 genotype/phenotype is covered when used to determine eligibility and dosing for Cerdelga in adult patients with Gaucher disease type 1; the test must be an FDA-cleared CYP2D6 pharmacogenetic test.

Results must document classification as extensive, intermediate, or poor metabolizer.

Provider Actions and Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is required for initial and continued therapy. Initial authorization may be granted for up to 6 months when the diagnostic and CYP2D6 testing criteria are met. Reauthorization (continuation of therapy) may be granted for up to 6 months when continuation criteria are met.

Initial authorization: up to 6 months when diagnostic and CYP2D6 testing criteria are met.
Reauthorization: up to 6 months when continuation criteria are met.

Documentation Required

Required Documentation

Provide documentation of a diagnosis of Gaucher disease type 1 confirmed by either an enzyme assay demonstrating deficiency of beta‑glucocerebrosidase activity or by genetic testing. Also provide documentation of CYP2D6 metabolizer status as detected by an FDA‑cleared test (extensive, intermediate, or poor metabolizer).

Documented diagnosis confirmed by enzyme assay or genetic testing.
CYP2D6 metabolizer status by an FDA‑cleared test (EM, IM, or PM).

Denial Risk

Non‑FDA Indications Not Covered

Use of Cerdelga for indications other than the FDA‑approved indication (adult Gaucher disease type 1 with CYP2D6 metabolizer status by an FDA‑cleared test) is not covered and is considered experimental/investigational and not medically necessary.

Non‑FDA indications are not covered.

Note

Provider Action Notes

Additional provider actions: ensure documentation submitted with the prior authorization request includes evidence supporting initial-approval criteria (diagnosis confirmation and CYP2D6 test results) and, for reauthorization, evidence the member is responding to therapy and not experiencing intolerable adverse events.

Include enzyme assay or genetic testing results and FDA‑cleared CYP2D6 test report with initial requests.
For reauthorization, include documentation that initial approval criteria continue to be met and that the patient is not experiencing inadequate response or intolerable adverse events.

Eligibility Requirements

To be eligible for coverage, the member must meet the document‑level requirements for Gaucher disease type 1 treatment. Specifically, the diagnosis must be confirmed by enzyme assay demonstrating beta‑glucocerebrosidase deficiency or by genetic testing, and CYP2D6 metabolizer status (extensive, intermediate, or poor) must be documented by an FDA‑cleared test. Authorization may be granted for up to 6 months when these requirements are met.

Background

Cerdelga (eliglustat) is indicated for the long‑term treatment of adults with Gaucher disease type 1 (GD1). Appropriate use depends on documented CYP2D6 metabolizer status determined by an FDA‑cleared test. When indicated and authorized, treatment duration is typically approved for up to 6 months. Dosing is guided by CYP2D6 status: 2 capsules daily for CYP2D6 extensive and intermediate metabolizers, and 1 capsule daily for CYP2D6 poor metabolizers.