Skip to main content

Type I Gaucher disease enzyme replacement therapy: imiglucerase (Cerezyme®), velaglucerase (Vpriv®) and telaglucerase (Elelyso®)

These services may or may not be covered by all CoOportunity Health plans. Please see your plan documents for your own coverage information. If there is a difference between this general information and your plan documents, your plan will be used to determine your coverage.

Administrative process

Therapies for Gaucher disease require prior authorization from Pharmacy Administration.

Coverage

Therapy for Gaucher’s disease is considered medically necessary for pediatric and adult patients with a confirmed diagnosis of Type I Gaucher disease resulting in one or more of the following conditions: moderate to severe anemia, thrombocytopenia with bleeding tendency, bone disease, significant hepatomegaly or splenomegaly.

For infused therapies: The labeled dosage is 60 units/kg administered every other week as a 60-minute intravenous infusion. However, after therapeutic goals are achieved, the lowest effective dose should be used.

Annual reauthorizations will require (1) a statement of progress against therapy goals which should include assessments of hemoglobin, platelet count, and liver and/or spleen volumes by MRI (when MRI is clinically indicated); and (2) for all regimens using more than 30 units/kg every other week, a statement of medical necessity indicating that the lowest effective dose to maintain therapeutic goals is being used. 

For eliglustat tartrate (Cerdelga): Utilization will be approved based on the patient’s CYP2D6 metabolism status and the FDA-approved regimen as follows:

  • Extensive metabolizers – Up to 84 mg twice daily.
  • Intermediate metabolizers – Up to 84 mg twice daily.
  • Poor metabolizers – Up to 84 mg once daily.

Annual reauthorizations will require a statement of progress against therapy goals which should include assessments of hemoglobin, platelet count, and liver and/or spleen volumes by MRI (when MRI is clinically indicated).

Definitions

Gaucher Disease is caused by a deficiency of the enzyme glucocerebrosidase, which helps the body metabolize the fatty substance glucocerebroside. Accumulation of glucocerebroside in various body organs prevents their normal function.

Three subtypes of Gaucher disease exist. Type I includes 99% of all Gaucher diagnoses and results in the non-neuropathic symptoms indicated above. Type II is characterized by neurologic symptoms and death within the first 18 months of life. Type III is similar to Type II but the onset of symptoms is later in life and the progression of disease over a longer period of time.

Cerezyme (imiglucerase) is indicated for long-term enzyme replacement therapy for pediatric and adult patients with a confirmed diagnosis of Type 1 Gaucher disease that results in one or more of the following conditions: anemia, thrombocytopenia, bone disease, or hepatomegaly or splenomegaly.

Vpriv (velaglucerase) is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for long-term enzyme replacement therapy (ERT) for pediatric and adult patients with type 1 Gaucher disease.

Elelyso (taliglucerase) is a hydrolytic lysosomal glucocerebroside-specific enzyme indicationed for long-term enzyme replacement therapy (ERT) indicated for Type 1 Gaucher’s disease in adults.

Ceredase (alglucerase), the original replacement therapy derived from human placental tissue, is not included in this policy as it is only available for limited use through compassionate use protocols. 

Cerdelga (eliglustat tartrate) is an oral agent approved for long term treatment of adult patients with Gaucher disease type 1 who are CHP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) as detected by an FDA-cleared test. The manufacturer has contracted with a central laboratory to provide metabolism assessments for all candidates for therapy.

Zavesca (miglustat) is not included in this policy as it is substrate reduction therapy not enzyme replacement therapy. It is a competitive and reversible inhibitor of glucosyltransferase which produces glucocerebroside. It does not require a prior authorization for use.

If available, codes are listed below for informational purposes only, and do not guarantee member coverage or provider reimbursement. The list may not be all-inclusive.

HCPCS Code

J1786 – Injection, imiglucerase, 10 units (Cerezyme)
J3060 – Injection, taliglucerase alfa, 10 units (Elelyso)
J3385 – Injection, velaglucerase alfa, 100 units (Vpriv)

NDC Codes

58468198301 – Cerezyme 200 unit single use solution
58468466301 – Cerezyme 400 unit single use solution
54092070104 – Vpriv 400 unit single dose solution
00069010601 – Elelyso 200 unit single use solution
5846802201 – Cerdelga carton of 4 packs of capsules (56 capsules total)
5846802201 – Cerdelga carton of 1 pack of capsules (14 capsules total)

ICD-9 Code Category

272 – Disorders of lipoid metabolism

CPT Copyright American Medical Association. All rights reserved. CPT is a registered trademark of the American Medical Association.

References

  1. Cerezyme Prescribing Information. Genzyme Corporation, Cambridge, MA. December 2012.
  2. Vpriv Prescribing Information. Shire Human Genetic Therapies, Inc. Cambridge, MA November 2013.
  3. Elelyso Prescribing Information. Pfizer Inc. New York, NY. May 2012.
  4. Cerdelga Prescribing Information. Genzyme Corporation. Cambridge, MA. August 2014
  5. Zimran A, Altarescu G, Philips M, et al., PhaseI/II and extension study of velaglucerase alfa (gene-activated human glucocerebrosidease) replacement therapy in adults with type 1 gaucher disease: 48 month experience. Blood 2010; 115 (23):4651.
  6. Anderson H, Charrow J, Kaplan P, et al., Individualization of long-term enzyme replacement therapy for Gaucher disease. Genetic Medicine 2005:7(2):105-110.
  7. Barton NW, Brady RO, Dambrosia JM, et al. Replacement therapy for inherited enzyme deficiency- macrophage-targeted glucocerebrodiase for Gaucher’s disease. N Engl J Med 1991; 324:1464-147. http://www.nejm.org/doi/full/10.1056/NEJM199105233242104
  8. Zimran, A. How I treat Gaucher disease. Blood 2011; 118:1463-1471. http://171.66.121.249/content/118/6/1463.full

This information is for most, but not all, CoOportunity Health plans. Please read your plan documents to see if your plan has limits or will not cover some items. If there is a difference between this general information and your plan documents, your plan documents will be used to determine your coverage.

CoOportunity Health has contracted with HealthPartners Administrators, Inc. to provide claims processing, medical management and certain other administrative services.