Subject: Gaucher disease medications

Drug

Cerdelga™  (eliglustat)

Cerezyme®  (imiglucerase)

Elelyso™  (taliglucerase alfa)

Vpriv™  (velaglucerase alfa)

Zavesca®  (miglustat)

Note: Precertification review for all medications except Cerdelga are handled through Aetna Specialty Precert Unit at 1-866-503-0857  See also Medical CPB Number: 0442

Policy:

Note: The provision of physician samples does not guarantee coverage under the provisions of the pharmacy benefit. All criteria below must be met in order to obtain coverage of each of these medications.

1. Precertification Criteria

Under some plans, including those that use an open or closed formulary, eliglustat (Cerdelga), imiglucerase (Cerezyme), taliglucerase alfa (Elelyso), velaglucerase alfa (VPRIV), and miglustat (Zavesca) are subject to precertification. If precertification requirements apply, Aetna considers these medications to be medically necessary for those members who meet the following precertification criteria:

 

Type 1 Gaucher disease, adult [Cerdelga, Cerezyme, Zavesca, Elelyso, VPRIV]

Initial Criteria

1. The member (≥ 18 years of age) has a documented diagnosis of type 1 Gaucher disease; and
2. Cerdelga only: The member has been tested to determine CYP2D6 genotype is one of the following: extensive metabolizer (EM), intermediate metabolize (IM), or poor metabolizer (PM); and
3. Zavesca only: Treatment with enzyme-replacement therapy (i.e. imiglucerase [Cerezyme], taliglucerase alfa [Elelyso], velaglucerase alfa [VPRIV]) was ineffective, not tolerated, or is contraindicated; and
4. The member has any of the following symptoms (a, b, c, d, or e):
   1. Moderate to severe anemia (hemoglobin ≤ 11.5 g/dL [adult women] or ≤ 12.5 g/dL [adult men] or ≤ 1.0 g/dL or more below the lower limit of normal for age and sex); or
   2. Significant hepatomegaly (liver size 1.25 or more times normal [1,750 cc in adults]) or splenomegaly (spleen size 5 or more times normal [875 cc in adults]); or
   3. Skeletal disease beyond mild osteopenia and Erlenmeyer flask deformity; or
   4. Symptomatic disease, including abdominal or bone pain, fatigue, exertional limitation, weakness, or cachexia; or
   5. Thrombocytopenia (platelet count less than or equal to 120,000/mm3).

 

Continuation Criteria

1. The member (≥ 18 years of age) has a documented diagnosis of type 1 Gaucher disease; and
2. There is clinical documentation indicating that there is disease stability or improvement (e.g., reduction in spleen or liver volume, normalized growth, MRI of spine/femurs, hematologic indices, reduced dependency on oxygen, or improvement in quality of life).

 

 

 

Type 1 Gaucher disease, pediatric [Cerdelga, Cerezyme, Zavesca, Elelyso, VPRIV]

Initial Criteria

1. The member (< 18 years of age) has a documented diagnosis of type 1 Gaucher disease; and
2. Cerdelga only: The member has been tested to the determine CYP2D6 genotype is one of the following: extensive metabolizer (EM), intermediate metabolizer (IM), or poor metabolizer (PM); and
3. Zavesca only: Treatment with enzyme-replacement therapy (i.e. imiglucerase [Cerezyme], taliglucerase alfa [Elelyso], velaglucerase alfa [VPRIV]) was ineffective, not tolerated, or is contraindicated.

 

Continuation Criteria:

1. The member (< 18 years of age) has a documented diagnosis of type 1 Gaucher disease; and
2. There is clinical documentation indicating that there is disease stability or improvement (e.g., reduction in spleen or liver volume, normalized growth, MRI of spine/femurs, hematologic indices, reduced dependency on oxygen, or improvement in quality of life).

 

 

 

Type 3 Gaucher disease, adult and pediatric [Cerezyme, Elelyso, VPRIV only]

Initial Criteria

1. The member has a documented diagnosis of type 3 Gaucher disease; and
2. The member has neurologic findings consistent with type 3 Gaucher disease, including encephalopathy, opthalmoplegia, progressive myoclonic epilepsy, cerebellar ataxia, spasticity, or dementia; and
3. The member has any of the following symptoms (a, b, c, d, or e):
   1. Moderate to severe anemia (hemoglobin ≤ 11.5 g/dL [adult women] or ≤ 12.5 g/dL [adult men] or ≤ 1.0 g/dL or more below the lower limit of normal for age and sex); or
   2. Significant hepatomegaly (liver size 1.25 or more times normal [1,750 cc in adults]) or splenomegaly (spleen size 5 or more times normal [875 cc in adults]); or
   3. Skeletal disease beyond mild osteopenia and Erlenmeyer flask deformity; or
   4. Symptomatic disease, including abdominal or bone pain, fatigue, exertional limitation, weakness, or cachexia; or
   5. Thrombocytopenia (platelet count less than or equal to 120,000/mm3).

 

Continuation Criteria

1. The member has a documented diagnosis of type 3 Gaucher disease; and
2. There is clinical documentation indicating that there is disease stability or improvement (e.g., reduction in spleen or liver volume, normalized growth, MRI of spine/femurs, hematologic indices, reduced dependency on oxygen, or improvement in quality of life).

 

*Note: Concomitant use of eliglustat (Cerdelga), imiglucerase (Cerezyme), taliglucerase alfa (Elelyso), velaglucerase alfa (VPRIV), and/or miglustat (Zavesca) for any indication is considered experimental/investigational.

 

 

 

According to the manufacturer, Cerdelga and Zavesca should be taken at the recommended common maintenance doses below. Quantities of these medications will be considered medically necessary as indicated in the table below.

Drug	Maximum Daily Dose	Dosage Strength	Quantity Limits
Cerdelga	168 mg	84 mg	Up to 60 capsules in 30 days
Zavesca	300 mg	100 mg	Up to 90 capsules in 30 days

Place of Service:

Outpatient

The above policy is based on the following references:

1. AHFS Drug Information® with AHFSfirstReleases®. (www.statref.com) American Society Of Health-System Pharmacists®, Bethesda, MD. Updated periodically.
2. DRUGDEX® System [Internet database]. Greenwood Village, CO: Thomson Micromedex. Updated periodically.
3. Drug Facts and Comparisons online. (www.drugfacts.com), Wolters Kluwer Health, St. Louis, MO. Updated periodically.
4. PDR® Electronic Library™ [Internet database]. Greenwood Village, CO: Thomson Micromedex. Updated periodically.
5. Cerdelga [prescribing information]. Cambridge, MA: Genzyme Corp.; Aug. 2014.
6. Cerezyme [prescribing information]. Cambridge, MA: Genzyme Corp.; March 2003.
7. Elelyso [prescribing information]. NY, NY: Pfizer, Inc.; June 2016.
8. VPRIV [prescribing information]. Lexington, MA: Shire Human Genetic Therapies, Inc.; April 2015.
9. Zavesca [prescribing information]. South San Francisco, CA: Actelion Pharmaceuticals US Inc.; Feb. 2014.
10. Andersson HC, Charrow J, Kaplan P, et al. Individualization of long-term enzyme replacement therapy for Gaucher disease. International Collaborative Gaucher Group U.S. Regional Coordinators. Genet Med. 2005;7(2):105.
11. Weinreb NJ, Aggio MC, Andersson HC, et al. Gaucher disease type 1: revised recommendations on evaluations and monitoring for adult patients. International Collaborative Gaucher Group (ICGG). Semin Hematol. 2004;41(4 Suppl 5):15.
12. Vellodi A, Tylki-Szymanska A, Davies EH, et al. Management of neuronopathic Gaucher disease: revised recommendations. European Working Group on Gaucher Disease. J Inherit Metab Dis. 2009;32(5):660.
13. Aetna Medical Clinical Policy Bulletin: Enzyme-replacement Therapy for Lysosomal Storage Disorders. Number 0442.

 

 

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.

March 24, 2021