CurrentMass General Brigham Health PlanPolicy N/A

Cerdelga (eliglustat) coverage and prior authorization

Defines prior authorization, quantity limit, specialty pharmacy requirement, and medical necessity criteria for Cerdelga (eliglustat) for Commercial/Exchange members of Mass General Brigham Health Plan.

Policy Summary

PayerMass General Brigham Health Plan

PolicyCerdelga (eliglustat) coverage and prior authorization

Policy CodePolicy N/A

Change TypeRoutine review — no material changes

Effective DateJul 1, 2025

Next Review DateJul 1, 2025

Key ActionSubmit prior authorization with documentation of Gaucher disease diagnosis and CYP2D6 metabolizer status by an FDA-cleared test.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

CommercialPlan applicability

12 monthsApproval length

PA + QLProgram types

SpecialtyFill setting

Coverage Criteria for Cerdelga (eliglustat)

Trek Health ingests and normalizes Transparency in Coverage data and payer policy updates to give provider organizations a clear view of how commercial reimbursement behaves across markets, payers, and services. Our platform transforms raw payer disclosures into structured intelligence that supports contract evaluation, payer negotiations, and service line strategy. By combining market benchmarks with ongoing policy visibility, Trek helps teams identify variability, risk, and opportunity in commercial reimbursement. The result is faster insight, stronger negotiating positions, and more informed financial decisions.

Diagnosis: Member has a diagnosis of Gaucher disease confirmed by enzyme assay demonstrating a deficiency of beta-glucocerebrosidase (glucosidase) enzyme activity or by genetic testing

Genotype: Submission of documentation showing that member is a CYP2D6 extensive metabolizer, an intermediate metabolizer, or a poor metabolizer as detected by an FDA-cleared test

Ultra-rapid metabolizers are not recommended due to inadequate concentrations

New-to-plan continuity: Members new to the plan within the past 90 days who are currently receiving the requested medication may be authorized when documentation of current treatment is provided (excluding samples or manufacturer patient assistance program supplies)

Continuation of Therapy

Reauthorization covered when ALL of the following are met:

Clinical response/tolerability: Member is not experiencing an inadequate response or any intolerable adverse events from therapy

Reauthorizations are granted for 12 months

Patients identified as CYP2D6 ultra-rapid metabolizers are not recommended for treatment with Cerdelga because they may not achieve adequate drug concentrations to produce a therapeutic effect. The FDA label also notes that a specific dosage cannot be recommended for patients whose CYP2D6 genotype is indeterminate (cannot be determined).

For prior authorization, the policy requires documentation of CYP2D6 metabolizer status as determined by an FDA-cleared test. Only members documented as extensive, intermediate, or poor metabolizers meet the genotype requirement for treatment with eliglustat; members who are ultra-rapid metabolizers or whose genotype is indeterminate should not be authorized for Cerdelga per the FDA indication and this policy.

Initial Authorization Requirements

Initial Authorization

Initial authorization criteria

Diagnosis confirmation: Documented Gaucher disease by enzyme assay or genetic testing

CYP2D6 testing: Documented CYP2D6 extensive, intermediate, or poor metabolizer status by an FDA-cleared test

Ultra-rapid metabolizers excluded

Reauthorization / Continuation Requirements

Reauthorization

Reauthorization requirement

Response and tolerability: Member is not experiencing inadequate response or intolerable adverse events

Reauthorizations are granted for 12 months and require documentation of continued clinical benefit and tolerability

Ongoing documentation: Clinical records documenting response and absence of intolerable adverse events must be submitted for reauthorization

Provider Actions and Documentation Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is required for Cerdelga (eliglustat). Include the diagnosis and CYP2D6 genotype status with the PA request to allow timely review.

Required with commercial/exchange plans
Program Type: Prior Authorization, Quantity Limit
Specialty pharmacy dispensing required

Step Therapy

No Step Therapy

There is no step therapy requirement for this medication.

Documentation Required

Required Clinical Documentation

Submit clinical documentation with the prior authorization request to support medical necessity.

Confirmation of Gaucher disease: enzyme assay showing beta‑glucocerebrosidase deficiency or genetic testing
Documentation of CYP2D6 status: report from an FDA‑cleared test indicating extensive, intermediate, or poor metabolizer

Documentation Required

New-to-Plan Treatment Documentation

Members new to the plan (within the past 90 days) who are currently receiving Cerdelga may be eligible for authorization. Provide documentation of current treatment (exclude samples or manufacturer patient assistance program supplies).

Member must be actively receiving the requested medication at the time of request
Do not accept samples or manufacturer patient assistance program medication as proof of current therapy

Denial Risk

Denial Risk for Missing Documentation

Missing diagnostic confirmation or CYP2D6 genotype documentation may result in denial of the prior authorization request. Ensure enzyme assay/genetic test results and FDA‑cleared CYP2D6 test report are included.

Absence of enzyme assay or genetic testing for Gaucher disease risks denial
Absence of an FDA‑cleared CYP2D6 test result (showing extensive, intermediate, or poor metabolizer) risks denial

Key Definitions

CYP2D6 metabolizer status — categories and relevance

Extensive metabolizer (normal)CYP2D6 extensive metabolizers — eligible phenotype for Cerdelga per FDA indication.

Intermediate metabolizerCYP2D6 intermediate metabolizers — eligible phenotype for Cerdelga per FDA indication.

Poor metabolizerCYP2D6 poor metabolizers — eligible phenotype for Cerdelga per FDA indication.

Ultra-rapid metabolizerCYP2D6 ultra-rapid metabolizers — not recommended for Cerdelga because they may not achieve adequate drug concentrations.

Indeterminate genotypeIndeterminate CYP2D6 genotype — a specific dosage cannot be recommended; not appropriate for selection of Cerdelga dosing.

Quantity Limit Designation

Cerdelga (eliglustat) quantity limit and dispensing

Program type designationQuantity Limit designation applied (policy lists Quantity Limit as a program type).

Per-period limitQuantity limit is applied per month (documented in quantity limits module).

Specialty dispensingMedication is designated specialty and must be filled at a contracted specialty pharmacy.

Site of Care and Fill Setting

Billing Rule

Specialty pharmacy dispensing required

Cerdelga is designated a specialty medication and must be dispensed through a contracted specialty pharmacy.

Fill must occur at a contracted specialty pharmacy per plan specialty limitations

Background

Gaucher disease type 1 is an inherited lysosomal storage disorder characterized by deficiency of the beta-glucocerebrosidase enzyme. Cerdelga (eliglustat) is a glucosylceramide synthase inhibitor approved for the long-term treatment of adults with Gaucher disease type 1.

The FDA-approved indication limits use to adults whose CYP2D6 metabolizer phenotype is classified as extensive, intermediate, or poor by an FDA-cleared test. This restriction reflects the drug’s pharmacokinetics: certain CYP2D6 genotypes (notably ultra-rapid metabolizers) can result in insufficient plasma concentrations and reduced likelihood of therapeutic benefit.