ModifiedCentenePolicy CP.PHAR.153

Eliglustat (Cerdelga) coverage for Type 1 Gaucher disease

Defines medical necessity, prior authorization, and continuation criteria for eliglustat (Cerdelga) for adults with type 1 Gaucher disease across Centene lines of business including Commercial, ICHRA/marketplace, and Medicaid.

Policy Summary

PayerCentene

PolicyEliglustat (Cerdelga) coverage for Type 1 Gaucher disease

Policy CodePolicy CP.PHAR.153

Change TypeMaterial operational updates

Effective DateFeb 1, 2016

Next Review DateN/A

Key ActionSubmit prior authorization with documentation of diagnosis, age, symptomatic disease, CYP2D6 genotype by an FDA‑cleared test, and dose within CYP2D6‑specific limits.

SourceLink

POLICY UPDATE CHANGES

Updated initial approval duration from 6 months to 12 months and added ICHRA (health insurance marketplace/ICHRA) as a line of business.

Updated initial approval duration from 6 months to 12 months.

Added ICHRA line of business.

12 monthsapproval duration (initial and continued)

84 mgproduct strength available

168 mg/daymax dose (EM/IM)

84 mg/daymax dose (PM)

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Coverage Criteria

Initial Therapy

Covered when ALL of the following are met for Initial Approval (Type 1 Gaucher Disease):

Initial Approval - GD1

Diagnosis of GD1 confirmed by one of the following: enzyme assay demonstrating a deficiency of beta-glucocerebrosidase activity OR DNA testing.
Age ≥ 18 years.
Symptoms: Member is symptomatic (e.g., anemia, thrombocytopenia, bone disease, hepatomegaly, splenomegaly).
CYP2D6 genotype: Member is positive for one of the following CYP2D6 genotypes as detected by an FDA-cleared test: Extensive metabolizer (EM), Intermediate metabolizer (IM), or Poor metabolizer (PM).
Monotherapy: Cerdelga is prescribed as monotherapy.
Dose limits: Dose does not exceed CYP2D6-specific maximums: CYP2D6 EMs and IMs ≤ 168 mg/day (84 mg PO BID); CYP2D6 PMs ≤ 84 mg/day (84 mg PO QD).

Initial Approval Duration: Initial authorization duration for eliglustat is 12 months.12 months

updated during annual review

Continuation Therapy

Covered when ALL of the following are met for Continued Therapy (Type 1 Gaucher Disease):

Continued Therapy - GD1

Prior therapy/continuity: Member meets one of the following: currently receiving medication via Centene benefit or previously met initial approval criteria; OR currently receiving medication and enrolled in a state/product with continuity of care regulations.
Response: Member is responding positively to therapy as evidenced by increased or stabilized platelet count or hemoglobin level, reduced or stabilized spleen or liver volume, or decreased bone pain.
Monotherapy: Cerdelga is prescribed as monotherapy.
Dose increase limits:

Not Medically Necessary / Not Authorized

Not authorized:

Not Authorized Uses: Non‑FDA approved indications not addressed in this policy are not authorized unless supported by sufficient documentation of efficacy and safety per applicable off-label use policies or evidence of coverage documents.

Contraindications / Exclusions: Situations meeting contraindications (for example: certain CYP inhibitor coadministration or hepatic impairment per CYP2D6 metabolizer status) are not appropriate for coverage as specified in contraindication guidance.

Non‑FDA approved indications that are not specifically addressed by this policy are not authorized unless the request is supported by applicable off‑label use policies or the member’s Evidence of Coverage. Refer to CP.CPA.09 for Commercial, HIM.PA.154 for Health Insurance Marketplace/ICHRA, and CP.PMN.53 for Medicaid when considering coverage for off‑label indications.

For Medicaid members, state Medicaid coverage provisions take precedence when they conflict with the coverage provisions in this clinical policy. If state rules differ from this policy, follow the applicable state Medicaid manual and its coverage determinations.

Certain situations are contraindications or medically inappropriate for eliglustat and are not appropriate for coverage. Contraindications are specified by CYP2D6 metabolizer status: for EMs, concomitant use of a strong or moderate CYP2D6 inhibitor together with a strong or moderate CYP3A inhibitor; moderate or severe hepatic impairment; or mild hepatic impairment when taking a strong or moderate CYP2D6 inhibitor. For IMs, concomitant strong/moderate CYP2D6 inhibitor with a strong/moderate CYP3A inhibitor, taking a strong CYP3A inhibitor, or any degree of hepatic impairment. For PMs, taking a strong CYP3A inhibitor or any degree of hepatic impairment are contraindications.

Initial Therapy Criteria

Initial Therapy

Covered when ALL of the following are met:

ALL of the following

Diagnosis of Type 1 Gaucher disease (GD1) confirmed by one of the following: enzyme assay demonstrating deficiency of beta-glucocerebrosidase (glucosidase) activity, OR DNA testing.
Age ≥ 18 years.
Member is symptomatic (examples: anemia, thrombocytopenia, bone disease, hepatomegaly, splenomegaly).
Member is positive for one of the following CYP2D6 genotypes as detected by an FDA-cleared test: Extensive metabolizer (EM), Intermediate metabolizer (IM), or Poor metabolizer (PM).

Continuation Therapy Criteria

Continuation Therapy

Continued therapy criteria for eliglustat (Cerdelga):

Eliglustat Continued Therapy - GD1: Refer to Continued Therapy criteria: current therapy or prior initial approval, positive clinical response, monotherapy, and dose within CYP2D6-specific maximums.

Approval duration: 12 months

Clinical response thresholds: Response may be evidenced by stabilized or increased hemoglobin or platelet counts, reduced or stabilized spleen or liver volume, or decreased bone pain. Clinical stability thresholds include: hemoglobin decrease <1.5 g/dL; platelet count decrease <25%; liver volume increase <20%; spleen volume increase <25%.

Dose limits on continuation: If increasing dose on continuation, new dose must not exceed CYP2D6-specific maximums: EM/IM 168 mg/day; PM 84 mg/day.

Provider Actions & Authorization

Prior Authorization

Submit PA with diagnosis, age, symptoms, CYP2D6 genotype, monotherapy, and dose limits

Prior authorization requests must include documentation that the member meets the clinical criteria: confirmed diagnosis of Type 1 Gaucher disease, age ≥18 years, symptomatic disease, CYP2D6 genotype (EM, IM, or PM) as determined by an FDA‑cleared test, and that Cerdelga is prescribed as monotherapy with dosing within CYP2D6‑specific limits.

Diagnosis confirmed by enzyme assay or DNA testing (see policy).
CYP2D6 phenotype reported from an FDA‑cleared test (EM, IM, or PM).
Dose within limits: EM/IM ≤168 mg/day; PM ≤84 mg/day.

Prior Authorization

Initial prior authorization duration updated to 12 months

The initial prior authorization term for eliglustat has been updated to 12 months per the 2Q 2026 policy revision.

Coding & Clinical Stability Thresholds

Clinical stability thresholds

HemoglobinDecrease < 1.5 g/dL

PlateletsDecrease < 25%

Liver volumeIncrease < 20%

Spleen volumeIncrease < 25%

Product and Quantity Limits

Eliglustat (Cerdelga) — dosing and phenotype limits

Product strength84 mg capsule

EM and IM dosing84 mg PO BID (maximum 168 mg/day)

PM dosing84 mg PO QD (maximum 84 mg/day)

Indication constraintUse in adults with GD1 who are CYP2D6 EM, IM, or PM as detected by an FDA‑cleared test

Definitions

GD1

DefinitionType 1 Gaucher disease (GD1): a heterogeneous disorder involving visceral organs, bone marrow, and bone resulting in anemia, thrombocytopenia, hepatomegaly, splenomegaly, and bone disease.

PopulationAdults with GD1 are eligible for eliglustat when CYP2D6 phenotype (EM/IM/PM) is confirmed by an FDA‑cleared test.

Clinical parametersHemoglobin, platelet count, liver and spleen volumes, and bone pain are used to assess therapeutic response.

CYP2D6 metabolizer phenotypes

EMExtensive metabolizer

IMIntermediate metabolizer

Background

Type 1 Gaucher disease (GD1) is a heterogeneous, inherited lysosomal storage disorder that primarily affects the visceral organs, bone marrow, and skeleton. Clinically, GD1 commonly presents with anemia, thrombocytopenia, hepatomegaly, splenomegaly, and various bone manifestations including bone pain and increased risk of fractures. Eliglustat (Cerdelga) is indicated for long‑term treatment of adults with GD1 who are CYP2D6 extensive, intermediate, or poor metabolizers as determined by an FDA‑cleared test.

Policy Summary

PayerCentene

PolicyEliglustat (Cerdelga) coverage for Type 1 Gaucher disease

Policy CodePolicy CP.PHAR.153

Change TypeMaterial operational updates

Effective DateFeb 1, 2016

Next Review DateN/A

Key ActionSubmit prior authorization with documentation of diagnosis, age, symptomatic disease, CYP2D6 genotype by an FDA‑cleared test, and dose within CYP2D6‑specific limits.

SourceLink

On This Page

If request is for a dose increase, the new dose must not exceed CYP2D6-specific maximums: CYP2D6 EMs and IMs ≤ 168 mg/day (2 capsules); CYP2D6 PMs ≤ 84 mg/day (1 capsule).

Continued Approval Duration: Continued authorization duration for eliglustat is 12 months.12 months

updated during annual review

Eliglustat (Cerdelga) is prescribed as monotherapy.

Dose does not exceed: CYP2D6 EMs and IMs — 168 mg (2 capsules) per day; CYP2D6 PMs — 84 mg (1 capsule) per day.

Approval duration: 12 months.

Prior Authorization

Provider Action: Prior Authorization & Documentation

Prior authorization is required. Submit documentation including diagnostic confirmation (enzyme assay or DNA testing), age, clinical symptoms, FDA-cleared CYP2D6 genotype test results, prescribed dose and regimen, and rationale for use as monotherapy. Documentation should demonstrate dosing consistent with CYP2D6 metabolizer status and support medical necessity. Cross-reference any applicable Centene policies for prior authorization procedures and genetic testing requirements. Failure to provide required documentation may result in denial or delayed processing.

Prior authorization required
Required documentation: diagnostic test results, CYP2D6 genotype test, clinical symptom documentation, prescribed dose/regimen, treatment rationale
Documentation expectations: clear test reports, dates, and prescriber notes; dose consistent with metabolizer status
Policy cross-reference: follow Centene prior authorization and genetic testing policy requirements

CONTINUATION CRITERIA

CONTINUATION CRITERIA — template and format updates applied during reviews:

Template updates: Continued therapy section formatting and templated language may be updated during periodic reviews; substantive coverage criteria remain as specified under Continued Therapy for GD1.

Operational note: Approval duration for continued therapy is set to 12 months per the most recent operational update.

Note

Refer to formulary/non‑formulary or off‑label policies when use not listed

If a requested use is not listed in this policy or the policy has not yet been updated for a recent label change, refer to the applicable formulary/no coverage, non‑formulary, or off‑label use policies for the relevant line of business as specified in the policy.

Formulary/no coverage and non‑formulary policy references are provided by line of business in the policy.
Off‑label use policy references are specified when the use is not covered here.

Documentation Required

Submit chart notes, labs, or other clinical information with PA

Providers must submit supporting clinical documentation (e.g., office chart notes, laboratory results, or other clinical information) demonstrating that the member meets all approval criteria.

Include documentation confirming diagnosis, CYP2D6 test results, symptom evidence, and current dosing.

Documentation Required

Ensure documentation aligns with Health Plan administrative policies

Follow Health Plan‑level administrative policies and procedures and ensure submitted documentation aligns with coverage determinations and the member's benefit documents.

Coverage decisions are subject to evidence of coverage, state/federal requirements, and Health Plan procedures.

Denial Risk

Denial triggers: unsupported off‑label use and contraindications

Coverage will not be authorized for non‑FDA approved indications unless there is sufficient documentation of efficacy and safety consistent with the applicable off‑label use policy; contraindications (eg, specific CYP inhibitor coadministration or hepatic impairment per metabolizer status) may also result in denial.

Non‑FDA indications require support per off‑label policies CP.CPA.09, HIM.PA.154, or CP.PMN.53 as applicable.
Contraindications vary by CYP2D6 phenotype (see Appendix C).

Denial Risk

Medicaid precedence: state provisions may trigger denial

For Medicaid members, state Medicaid coverage provisions take precedence; if state rules conflict with this clinical policy and disallow coverage, the request may be denied in accordance with the state Medicaid manual.

Check the applicable state Medicaid manual for any differing coverage provisions.

Health Plan

Health Plan definitionA health plan that has adopted this clinical policy and that is operated or administered, in whole or in part, by Centene Management Company, LLC, or affiliates.

PurposeProvides guidance on medical necessity to assist in coverage decisions and administering benefits.

Not a contractThis clinical policy does not constitute a contract or guarantee of payment; coverage is subject to benefit documents and legal/regulatory requirements.