CurrentNeighborhood Health Plan of Rhode IslandPolicy 2050-A

Cerdelga 2050 A Sgm P2023

Policy defines prior authorization criteria for coverage of Cerdelga (eliglustat) for long-term treatment of adult patients with Gaucher disease type 1, including required diagnostic testing and metabolizer status; continuation criteria and exclusions for non-approved uses are included.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyCerdelga 2050 A Sgm P2023

Policy CodePolicy 2050-A

Change TypeNo material change

Effective Date

Next Review Date

Key ActionProviders must submit beta-glucocerebrosidase enzyme assay or genetic testing results supporting diagnosis and results of an FDA-cleared CYP2D6 test to initiate prior authorization review.

SourceLink

POLICY UPDATE CHANGES

No material clinical/coverage changes

1Covered Indication(s)

3Required Documentation Items

12Authorization Duration (months)

Coverage Summary

Coverage stance: covered_with_criteria for Cerdelga (eliglustat).

Scope summary: Policy defines prior authorization criteria for coverage of Cerdelga (eliglustat) for long-term treatment of adult patients with Gaucher disease type 1, requiring diagnostic confirmation by beta-glucocerebrosidase enzyme assay or genetic testing and documentation of CYP2D6 metabolizer status (EM, IM, or PM) prior to approval. Continuation criteria and exclusions for non‑approved uses are included.

Initial Therapy Criteria

Initial Authorization for Gaucher disease type 1

Authorization of 12 months may be granted when ALL of the following criteria are met:

ALL of the following

Diagnosis confirmed: Diagnosis of Gaucher disease confirmed by enzyme assay demonstrating a deficiency of beta-glucocerebrosidase (glucosidase) enzyme activity or by genetic testing
CYP2D6 metabolizer status: Member is a CYP2D6 extensive metabolizer (EM), intermediate metabolizer (IM), or poor metabolizer (PM) as detected by an FDA-cleared test

Required Documentation to Initiate Prior Authorization

Submission of the following information is necessary to initiate the prior authorization review:

Required documentation

Diagnosis documentation: Beta-glucocerebrosidase enzyme assay or genetic testing results supporting diagnosis
CYP2D6 test results: Results of the CYP2D6 test

Prior Authorization

Prior authorization required with documentation

Providers must submit documentation to initiate prior authorization review. Specifically, submit results confirming Gaucher disease (beta‑glucocerebrosidase enzyme assay or genetic testing) and results of an FDA‑cleared CYP2D6 test so the member’s metabolizer status can be determined.

Beta‑glucocerebrosidase enzyme assay results or genetic testing confirming Gaucher disease
Results of an FDA‑cleared CYP2D6 test showing metabolizer status (EM, IM, or PM)

Continuation Therapy Criteria

Continuation of Therapy

Authorization of 12 months may be granted for continued treatment when ALL of the following criteria are met:

Continuation criteria

Meets initial criteria: Member meets the criteria for initial approval
Clinical response: Member is not experiencing an inadequate response or any intolerable adverse events from therapy

Documentation Required

Documentation for continuation

For renewal, document that the member continues to meet the initial approval criteria and that therapy is tolerated and effective. Specifically, confirm ongoing fulfillment of the initial diagnostic and CYP2D6 metabolizer criteria and state that the member is not experiencing an inadequate response or any intolerable adverse events from eliglustat.

Confirmation that initial approval criteria remain met (diagnosis and CYP2D6 metabolizer status)
Statement that there is no inadequate response to therapy
Statement that there are no intolerable adverse events from therapy

Not Medically Necessary / Exclusions

Exclusions / Not medically necessary

CYP2D6 URM: Patients who are CYP2D6 ultra-rapid metabolizers (URMs): may not achieve adequate concentrations of Cerdelga to achieve a therapeutic effect
Indeterminate genotype: Patients whose CYP2D6 genotype cannot be determined (indeterminate metabolizers): a specific dosage cannot be recommended
Non-approved indications: All other indications (outside FDA-approved indications and compendial uses) are considered experimental/investigational and not medically necessary

Denial Risk

Denial risk for excluded metabolizer statuses and non‑approved uses

Requests for patients who are CYP2D6 ultra‑rapid metabolizers, whose CYP2D6 genotype cannot be determined (indeterminate), or for indications outside the FDA‑approved/compendial uses are not considered medically necessary and are likely to be denied.

CYP2D6 ultra‑rapid metabolizers (URMs) — may not achieve adequate drug concentrations
Indeterminate CYP2D6 genotype — dosing cannot be recommended
All other indications outside the FDA‑approved indications/compendial uses (experimental/investigational)

Clinical Evidence

Evidence: Primary reference: Cerdelga [package insert], Genzyme Corporation; July 2021.

Note: Per FDA labeling and the package insert, CYP2D6 metabolizer status affects eliglustat metabolism and dosing; ultra-rapid metabolizers may not achieve adequate drug concentrations and indeterminate genotypes have no specific dosage recommendation (see Definitions for CYP2D6 metabolizer statuses).

Background & Definitions

Background: Cerdelga (eliglustat) is FDA‑approved for the long-term treatment of adult patients with Gaucher disease type 1 who are CYP2D6 extensive, intermediate, or poor metabolizers as detected by an FDA-cleared test.

Prior to approval, the policy requires confirmation of diagnosis by beta-glucocerebrosidase enzyme assay or genetic testing and results of an FDA‑cleared CYP2D6 test to establish metabolizer status.

Definitions

CYP2D6 metabolizer statusesGenetic categorizations (extensive, intermediate, poor, ultra-rapid, indeterminate) determined by an FDA-cleared test that affect eliglustat metabolism and dosing/efficacy. URMs may not achieve adequate concentrations; indeterminate genotypes prevent dosage recommendation.