Subject: Cerdelga SGM 2050-A P2025

Drug

CERDELGA  (eliglustat)

Policy:

Indications

The indications below including FDA-approved indications and compendial uses are considered a covered benefit provided that all the approval criteria are met and the member has no exclusions to the prescribed therapy.

FDA-approved Indications¹

Cerdelga is indicated for the long-term treatment of adult patients with Gaucher disease type 1 (GD1) who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) as detected by an FDA-cleared test.

Limitations of Use

Patients who are CYP2D6 ultra-rapid metabolizers (URMs) may not achieve adequate concentrations of Cerdelga to achieve a therapeutic effect. A specific dosage cannot be recommended for those patients whose CYP2D6 genotype cannot be determined (indeterminate metabolizers).

All other indications are considered experimental/investigational and not medically necessary.

Documentation

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

• Beta-glucocerebrosidase (glucosidase) enzyme assay or genetic testing results supporting diagnosis, and
• The results of the CYP2D6 test.

Prescriber Specialties

This medication must be prescribed by or in consultation with a physician who specializes in the treatment of metabolic disease and/or lysosomal storage disorders.

Coverage Criteria

Gaucher disease type 1¹

Authorization of 12 months may be granted for treatment of Gaucher disease type 1 when all of the following criteria are met:

• Member is 18 years of age or older.
• Diagnosis of Gaucher disease was confirmed by enzyme assay demonstrating a deficiency of beta-glucocerebrosidase (glucosidase) enzyme activity or by genetic testing.
• Member is a CYP2D6 extensive metabolizer, an intermediate metabolizer, or a poor metabolizer as detected by an FDA-cleared test.

Continuation of Therapy

Gaucher disease type 1¹

Authorization of 12 months may be granted for continued treatment of an indication listed in the coverage criteria section when all of the following criteria are met:

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

Place of Service:

Outpatient

The above policy is based on the following references:

1. Cerdelga [package insert]. Cambridge, MA: Genzyme Corporation; January 2024.

Copyright Aetna Inc. All rights reserved. Pharmacy Clinical Policy Bulletins are developed by Aetna to assist in administering plan benefits and constitute neither offers of coverage nor medical advice. This Clinical Policy Bulletin contains only a partial, general description of plan or program benefits and does not constitute a contract. Aetna does not provide health care services and, therefore, cannot guarantee any results or outcomes. Participating providers are independent contractors in private practice and are neither employees nor agents of Aetna or its affiliates. Treating providers are solely responsible for medical advice and treatment of members. This Clinical Policy Bulletin may be updated and therefore is subject to change.

July 22, 2025