Prior Auth Protocol

FDA Approved Indications:

CEREZYME

Long-term enzyme replacement therapy for pediatric and adult patients with a confirmed diagnosis of Type I Gaucher disease that results in one or more of the following conditions:

Anemia
Thrombocytopenia
Bone disease
Hepatomegaly or splenomegaly

VPRIV

Long-term enzyme replacement therapy (ERT) for patients with Type I Gaucher disease

ELELYSO

Long-term enzyme replacement therapy (ERT) for adults and pediatric patients with a confirmed diagnosis of Type I Gaucher disease

Health Net Approved Indications and Usage Guidelines:

Diagnosis of Type 1 or Type 3 Gaucher disease

AND

Diagnosis confirmed by measurement of beta-glucocerebrosidase activity (in leukocytes or skin fibroblasts) of less than 30% of normal values OR deoxyribonucleic acid (DNA) testing (mutations in the glucocerebrosidase gene)

AND

Patient exhibits clinical signs and symptoms of the disease, including anemia, thrombocytopenia, skeletal disease, or visceromegaly (liver or spleen enlargement).

Coverage is Not Authorized For:

Non-FDA approved indications, which are not listed in the Health Net Approved Indications and Usage Guidelines section, unless there is sufficient documentation of efficacy and safety in the published literature.

General Information:

Physicians must complete and sign the Start Form (VPRIV Prescription and OnePath^SM Start Form) as well as the Statement of Medical Necessity form. Call a OnePath^SM Case Manager at 1-866-888-0660 to access the necessary forms.
Enzyme replacement therapy may have beneficial palliative effects in Type 2 disease, but does not alter the outcome and is not generally used.
According to the European consensus guidelines revised recommendations on the management of neuronopathic Gaucher disease by Vellodi et al: (1) there is clear evidence in most patients that enzyme replacement therapy (ERT) ameliorates systemic involvement in non-neuronopathic (Type 1) as well as chronic neuronopathic Gaucher disease (Type 3); enhancing quality of life (2) There is no evidence that ERT has reversed, stabilized or slowed the progression of neurological involvement; (3) In patients with established acute neuronopathic Gaucher disease (Type 2), enzyme replacement therapy has had little effect on the progressively downhill course. It has merely resulted in prolongation of pain and suffering.
In a study by Altarescu et al, enzyme replacement therapy with Ceredase/Cerezyme reversed the systemic manifestations (hemoglobin levels, platelet counts, liver and spleen volumes) in 19 patients with type 3 Gaucher disease. There was no evidence that improvement in the systemic disease manifestations led to early cognitive improvement or altered the neurologic course. Doses of Ceredase/Cerezyme used ranged from 120 unit/kg - 480 units/kg per month with the average being 120 units/kg every 2 weeks.
There is currently insufficient clinical evidence that supports the combination use of enzyme replacement therapy with Zavesca^R (miglustat)

Therapeutic Alternatives:

Drug	Dosing Regimen	Dose Limit/ Maximum Dose
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* Requires Prior Authorization

Recommended Dosing Regimen and Authorization Limit:

Drug	Dosing Regimen	Authorization Limit
Cerezyme	2.5 units/kg via IV infusion 3 times weekly to 60 units/kg once every 2 weeks	6 months or to member's renewal period, whichever is sooner.
VPRIV	60 units/kg via IV infusion every other week	6 months or to member's renewal period, whichever is sooner.
Eleyso	60 units/kg via IV infusion every other week	6 months or to member's renewal period, whichever is sooner.

Product Availability:

Cerezyme: 200 units and 400 units per vial

VPRIV: 400 units per vial
Elelyso: 200 units per vial

References:

1. Cerezyme [Product information] Cambridge, MA: Genzyme Corporation; March 2003.

2. VPRIV [Prescribing Information] Cambridge, MA: Shire Human Genetics Therapies, Inc; April 2015.

3. Elelyso [Prescribing Information] New York, NY: Pfizer Labs; August 2014.

4. Altarescu G, Hill S, Wiggs E, et al. The efficacy of enzyme replacement therapy in patients with chronic neuronopathic Gaucher`s disease. J Pediatr. 2001;138:539-547.

5. Vellodi A, Tylki-Szymanska A, Davies E, et al. Management of neuronopathic Gaucher disease: Revised recommendations. J Inherit Metab Dis. 2009;32:660-664.

6. Hughes, D. Gaucher disease: Treatment (UpToDate website). Available at www.uptodate.com. Accessed May 22, 2015.

7. Cerezyme. In: DrugPoints^R System [Internet database]. Greenwood Village, Colo: Thomson Healthcare. Updated periodically. Accessed May 22, 2015.

8. VPRIV. In: DrugPoints^R System [Internet database]. Greenwood Village, Colo: Thomson Healthcare. Updated periodically. Accessed May 22, 2015.

9. Elelyso. Micromedex^R Healthcare Series [Internet database]. Greenwood Village, Colo: Thomson Healthcare. Updated periodically. Accessed May 22, 2015.

10. Cerzyme. Amercian Hospital Formulary Service Drug Information. Available at: http://www.medicinescomplete.com/mc/ahfs/current/. Accessed May 22, 2015.

11. VPRIV. Amercian Hospital Formulary Service Drug Information. Available at: http://www.medicinescomplete.com/mc/ahfs/current/. Accessed May 22, 2015.

12. Clinical Pharmacology Web site. Available at: http://clinicalpharmacology-ip.com/. Accessed May 22, 2015.