CurrentmhnPolicy CP.PHAR.153

Eliglustat (Cerdelga) for Type 1 Gaucher Disease

Defines medical necessity criteria, dosing limits, contraindications, and prior-authorization documentation requirements for eliglustat (Cerdelga) for adults with type 1 Gaucher disease for health plans affiliated with Centene (Commercial, HIM, Medicaid).

Policy Summary

Payermhn

PolicyEliglustat (Cerdelga) for Type 1 Gaucher Disease

Policy CodePolicy CP.PHAR.153

Change TypeNo material changes

Effective DateFeb 1, 2016

Next Review Date

Key ActionProvider must submit documentation (office notes, lab results, DNA testing or enzyme assay results) confirming GD1 diagnosis and CYP2D6 metabolizer status detected by an FDA-cleared test.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

18 monthsMaximum combined approval durations shown (6 month initial + 12 month continuation)

84/168Available capsule strength and maximum daily doses by genotype

≥18Minimum patient age for coverage

FDA-approvedCoverage limited to GD1 adults with appropriate CYP2D6 metabolizer status

Medical Necessity Criteria

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Initial Approval — Type 1 Gaucher Disease

Cerdelga is medically necessary when ALL of the following are met:

ALL of the following

Diagnosis of type 1 Gaucher disease (GD1) confirmed by EITHER enzyme assay demonstrating a deficiency of beta-glucocerebrosidase activity OR DNA testing
Either enzyme assay OR DNA testing acceptable
Patient age is ≥ 18 years
Member is symptomatic (examples provided: anemia, thrombocytopenia, bone disease, hepatomegaly, splenomegaly)
CYP2D6 genotype
- Extensive metabolizer (EM) as detected by an FDA-cleared test
- Intermediate metabolizer (IM) as detected by an FDA-cleared test
- Poor metabolizer (PM) as detected by an FDA-cleared test
Cerdelga is prescribed as monotherapy
Dose limits
- For CYP2D6 EMs and IMs: total daily dose must not exceed 168 mg (84 mg PO BID; 2 capsules per day)≤ 168 mg/day
- For CYP2D6 PMs: total daily dose must not exceed 84 mg (84 mg PO QD; 1 capsule per day)≤ 84 mg/day

(Approval duration: 6 months)

Initial Approval — Other diagnoses/indications

If request is for other indications or recent label changes, follow formulary/non-formulary or off-label policies:

ANY of the following

If drug had recent label change not yet reflected, refer to the relevant no coverage or non-formulary policy for the line of business (see related_policies list)
If requested use is not listed under section III and above does not apply, refer to the off-label use policy for the relevant line of business (see related_policies list)

(See related policies CP.CPA.190, HIM.PA.33, CP.PMN.255, HIM.PA.103, CP.PMN.16, CP.CPA.09, HIM.PA.154, CP.PMN.53 as applicable)

Continuation (Renewal) Approval — Type 1 Gaucher Disease

Cerdelga is medically necessary for continuation when ALL of the following are met:

ALL of the following

Continuation eligibility
- Member currently receiving medication via Centene benefit or has previously met initial approval criteria
- Member is currently receiving medication and is enrolled in a state/product with continuity of care regulations (refer to state addendums CC.PHARM.03A and CC.PHARM.03B)
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
Cerdelga is prescribed as monotherapy

Not Authorized / Exclusions

Coverage is NOT authorized for the following:

ANY of the following

Non-FDA approved indications that are not addressed in this policy, unless there is sufficient documentation per the applicable off-label use policies or evidence of coverage documents

(See off-label use policies CP.CPA.09, HIM.PA.154, CP.PMN.53 for allowable exceptions)

Prior Authorization & Documentation Requirements

Prior Authorization

Prior authorization criteria for initiation

Prior authorization will be approved only when all initial approval criteria are met: patient is age ≥ 18, has symptomatic type 1 Gaucher disease confirmed by enzyme assay or DNA testing, has a CYP2D6 metabolizer status (EM, IM, or PM) determined by an FDA-cleared test, Cerdelga is prescribed as monotherapy, and the total daily dose does not exceed genotype-specific limits (≤168 mg/day for EMs and IMs; ≤84 mg/day for PMs).

Diagnosis confirmed by enzyme assay demonstrating beta-glucocerebrosidase deficiency OR DNA testing (see chunk 5).
CYP2D6 metabolizer status must be EM, IM, or PM as detected by an FDA-cleared test (see chunk 5 and 2).
Dose limits: EMs/IMs ≤ 168 mg/day (84 mg PO BID); PMs ≤ 84 mg/day (84 mg PO QD) (see chunk 6 and 15).
Cerdelga must be prescribed as monotherapy (see chunk 5).

Documentation Required

Submit diagnostic confirmation and clinical documentation

Submit supporting clinical documentation with the prior authorization request, including office/chart notes, laboratory results, and the diagnostic test demonstrating GD1 (enzyme assay or DNA testing) plus documentation of CYP2D6 metabolizer status from an FDA-cleared test.

Office notes or chart documentation confirming symptoms (e.g., anemia, thrombocytopenia, bone disease, hepatomegaly, splenomegaly) (see chunk 5).
Enzyme assay demonstrating deficiency of beta-glucocerebrosidase OR DNA testing to confirm GD1 (see chunk 5).
Result from an FDA-cleared test showing CYP2D6 metabolizer status (EM/IM/PM) (see chunk 5 and 2).

Documentation Required

Demonstrate clinical response for renewal

For renewal/continuation, provide documentation that the member is responding positively to therapy using clinical measures such as improved or stabilized hemoglobin or platelet counts, reduced or stabilized spleen or liver volume, or decreased bone pain.

Evidence of increased or stabilized platelet count or hemoglobin level, reduced or stabilized spleen or liver volume, or decreased bone pain (see chunk 8 and 13).
If request is for a dose increase, ensure new dose does not exceed genotype-specific renewal limits (EMs/IMs ≤ 168 mg/day; PMs ≤ 84 mg/day) (see chunk 8).

Denial Risk

Denial risk for indeterminate genotype or contraindicated concomitant drugs

Requests may be denied if the CYP2D6 genotype is indeterminate, if the member is a CYP2D6 ultra-rapid metabolizer, or if contraindications (including specific concomitant CYP3A/CYP2D6 inhibitor combinations or any/moderate–severe hepatic impairment per genotype) preclude safe dosing.

CYP2D6 ultra-rapid metabolizers may not achieve therapeutic concentrations; indeterminate genotype has no recommended dosage (see chunk 3 and 14).
Contraindications vary by genotype (e.g., EMs: strong/moderate CYP2D6 inhibitor with strong/moderate CYP3A inhibitor; IMs/PMs: restrictions with CYP3A or CYP2D6 inhibitors and hepatic impairment) (see chunk 12).

Monitoring Parameters & Dose Limits

Stability and Dosing Thresholds

Hemoglobin stability threshold< 1.5 g/dL decrease

Platelet count stability threshold< 25% decrease

Liver volume stability threshold< 20% increase

Spleen volume stability threshold< 25% increase

Dose limit for EMs/IMs≤ 168 mg/day (84 mg PO BID)

Dose limit for PMs≤ 84 mg/day (84 mg PO QD)

Contraindications vary by CYP2D6 metabolizer status and hepatic function and should be reviewed when assessing safety. For extensive metabolizers (EMs), eliglustat is contraindicated when the patient is taking a strong or moderate CYP2D6 inhibitor concomitantly with a strong or moderate CYP3A inhibitor, in moderate or severe hepatic impairment, and in mild hepatic impairment when a strong or moderate CYP2D6 inhibitor is being taken.

For intermediate metabolizers (IMs), eliglustat is contraindicated when the patient is taking a strong or moderate CYP2D6 inhibitor concomitantly with a strong or moderate CYP3A inhibitor, when the patient is taking a strong CYP3A inhibitor, and with any degree of hepatic impairment.

For poor metabolizers (PMs), eliglustat is contraindicated when the patient is taking a strong CYP3A inhibitor and with any degree of hepatic impairment. Reference these genotype- and hepatic function–specific contraindications when evaluating concomitant medications and liver status to determine whether eliglustat can be used safely.

Clinical Background

Eliglustat (Cerdelga) is an oral glucosylceramide synthase inhibitor approved for long-term treatment of adult patients with type 1 Gaucher disease (GD1) who have a determined CYP2D6 metabolizer status (EM, IM, or PM) as detected by an FDA-cleared test. CYP2D6 genotype is required to guide dosing and ensure appropriate exposure.

Therapeutic response to eliglustat should be monitored with routine clinical parameters including hemoglobin level, platelet count, liver and spleen volume, and bone pain, since GD1 commonly involves visceral organs, bone marrow, and bone. Clinical trial and policy definitions of stability include thresholds such as hemoglobin decrease < 1.5 g/dL, platelet decrease < 25%, liver volume increase < 20%, and spleen volume increase < 25% as examples of acceptable stability.

Dosing is genotype-directed: for CYP2D6 EMs and IMs the usual regimen is 84 mg PO BID (maximum 168 mg/day); for CYP2D6 PMs the regimen is 84 mg PO QD (maximum 84 mg/day).

OpenPayer

Eliglustat (Cerdelga) for Type 1 Gaucher Disease

Medical Necessity Criteria

Product Availability / Coding

Prior Authorization & Documentation Requirements

Monitoring Parameters & Dose Limits

Clinical Background

Defined Terms

Policy Dates

Eliglustat (Cerdelga) for Type 1 Gaucher Disease

Medical Necessity Criteria

Product Availability / Coding

Prior Authorization & Documentation Requirements

Monitoring Parameters & Dose Limits

Clinical Background

Defined Terms

Cross‑Reference Policies

Policy Dates