Pharmacy Prior Authorization Criteria

Gaucher Disease Agents

Indications for Prior Authorization

Cerezyme (imiglucerase for injection)

• For diagnosis of Type 1 Gaucher Disease
  Indicated for treatment of adults and pediatric patients 2 years of age and older with Type 1 Gaucher disease that results in one or more of the following conditions:

  - anemia - thrombocytopenia - bone disease - hepatomegaly or splenomegaly

Elelyso (taliglucerase alfa) for injection

• For diagnosis of Type 1 Gaucher Disease
  Indicated for the treatment of patients 4 years and older with a confirmed diagnosis of Type 1 Gaucher disease.

VPRIV (velaglucerase alfa for injection)

• For diagnosis of Type 1 Gaucher Disease
  Indicated for long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease.

Cerdelga (eliglustat)

• For diagnosis of Type 1 Gaucher Disease
  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).

Zavesca (miglustat), Yargesa (miglustat)

• For diagnosis of Type 1 Gaucher Disease
  Indicated as monotherapy for the treatment of adult patients with mild to moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option (e.g., due to allergy, hypersensitivity, or poor venous access).

Criteria

Cerezyme, Elelyso, or VPRIV

Prior Authorization

Length of Approval: 12 Month(s)

• Diagnosis of Type 1 Gaucher disease
AND
• Patient has evidence of symptomatic disease (e.g., moderate to severe anemia [A], thrombocytopenia [B], bone disease [C], hepatomegaly [D], or splenomegaly [D])
AND
• One of the following:
• Patient is 4 years of age or older (applies to Elelyso and VPRIV only)
OR
• Patient is 2 years of age or older (applies to Cerezyme only)

Cerdelga

Prior Authorization

Length of Approval: 12 Month(s)

• Diagnosis of Type 1 Gaucher disease
AND
• Patient is an extensive metabolizer (EM), intermediate metabolizer (IM), or poor metabolizer (PM) of cytochrome P450 enzyme (CYP) 2D6 as detected by an FDA-cleared test
AND
• Patient is 18 years of age or older

Brand Zavesca, Generic miglustat, or Yargesa

Prior Authorization

Length of Approval: 12 Month(s)
For diagnosis of Type 1 Gaucher Disease

• Diagnosis of mild to moderate Type 1 Gaucher disease
AND
• Patient is 18 years of age or older

Brand Zavesca, Generic miglustat

Prior Authorization (Initial Authorization)

Length of Approval: 6 Month(s)
For diagnosis of Niemann-Pick disease type C (NPC) (off-label) [E]

• Diagnosis of Niemann-Pick disease type C (NPC)
AND
• Requested drug will be used in combination with Miplyffa (arimoclomol)
AND
• Prescribed by or in consultation with a specialist knowledgeable in the treatment of Niemann-Pick disease type C

Brand Zavesca, Generic miglustat

Prior Authorization (Reauthorization)

Length of Approval: 12 Month(s)
For diagnosis of Niemann-Pick disease type C (NPC) (off-label) [E]

• Patient demonstrates positive clinical response to therapy
AND
• Requested drug will be used in combination with Miplyffa (arimoclomol)

P & T Revisions

2025-01-16, 2024-11-11, 2024-02-20, 2023-11-23, 2023-02-17, 2022-05-23, 2022-02-18, 2021-09-27, 2021-05-19, 2021-02-03, 2020-01-29

References

1. Cerezyme Prescribing Information. Genzyme Corporation. Cambridge, MA. December 2022.
2. Elelyso Prescribing Information. Pfizer, Inc. New York, NY. May 2023.
3. VPRIV Prescribing Information. Takeda Pharmaceuticals U.S.A., Inc. Lexington, MA. September 2021.
4. Cerdelga Prescribing Information. Genzyme Corporation. Cambridge, MA. December 2023.
5. Zavesca Prescribing Information. Actelion Pharmaceuticals US, Inc. Titusville, NJ. August 2022.
6. Pastores GM, Weinreb NJ, Aerts H, et al. Therapeutic goals in the treatment of Gaucher disease. Semin Hematol. 2004;41(4 Suppl 5):4-14.
7. Weinreb NJ, Aggio MC, Andersson HC, et al. Gaucher disease type 1: revised recommendations on evaluations and monitoring for adult patients. Semin Hematol. 2004;41(suppl 5):15-22.
8. Weinreb N, Taylor J, Cox T, et al. A benchmark analysis of the achievement of therapeutic goals for type 1 Gaucher disease patients treated with imiglucerase. Am J Hematol. 2008;83:890-895.
9. Hollak CE, vom Dahl S, Aerts JM, et al. Force majeure: therapeutic measures in response to restricted supply of imiglucerase (Cerezyme) for patients with Gaucher disease. Blood Cells Mol Dis. 2010;44(1):41-7.
10. Per clinical consult with geneticist, November 11, 2010.
11. Yargesa Prescribing Information. Edenbridge Pharmaceuticals LLC. Parsippany, NJ. October 2023.
12. Miplyffa Prescribing Information. Zevra Therapeutics, Inc. FL 34747. September 2024.
13. Mengel E, Patterson MC, Da Riol RM et al. Efficacy and safety of arimoclomol in Niemann-Pick disease type C: Results from a double-blind, randomised, placebo-controlled, multinational phase 2/3 trial of a novel treatment. J Inherit Metab Dis. 2021 Nov;44(6):1463-1480. doi: 10.1002/jimd.12428. Epub 2021 Sep 7.
14. FDA Review: Miplyffa. Food and Drug Administration Web Site. 2024. http://www.accessdata.fda.gov. Accessed November 4, 2024.

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End Notes

1. Goals of treatment with anemia are to increase hemoglobin to greater than or equal to 12.0 g/dL for males (greater than 12 years of age), and to greater than or equal to 11.0 g/dL for both children (less than or equal to 12 years of age) and females (greater than 12 years of age). [6, 8]
2. Moderate thrombocytopenia is defined as a platelet count of 60,000 to 120,000/microliter. A platelet count of 120,000/microliter to meet the criterion of thrombocytopenia is based on the upper end of the range that defines moderate thrombocytopenia. [6]
3. In bone disease, the goal is to lessen or eliminate bone pain and prevent bone crises. Bone disease can be diagnosed using MRI, bone scan, and X-ray. [6-8]
4. Hepatomegaly is defined as a liver mass of greater than 1.25 times normal value. Splenomegaly is defined as a splenic mass greater than the normal, and moderate splenomegaly is considered a spleen volume of greater than 5 and less than or equal to 15 times normal. [6]
5. Criteria is here to support the off-label use of miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in combination with Miplyffa as per Miplyffa FDA labelling. [12-14]

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Revision History

• 2025-01-16: Administrative update to indicate second criteria box for NPC indication is reauthorization criteria.
• 2024-11-11: Criteria added to support the off-label use of miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in combination with Miplyffa.
• 2024-02-20: 2024 annual review: no criteria changes. Updated end notes and background.
• 2023-11-23: Added branded generic for Zavesca 100mg capsules.
• 2023-02-17: Annual review - no criteria changes.
• 2022-05-23: Update to remove Cerdelga reauthorization criteria and Update to Zavesca criteria
• 2022-02-18: Annual review: added age criterion for Elelyso, VPRIV, Cerezyme, and Zavesca/miglustat to align with the labels.
• 2021-09-27: Addition of EHB formulary to guideline, no changes to criteria
• 2021-05-19: Addition of EHB formulary to guideline, no changes to criteria
• 2021-02-03: Annual review: Background updates.
• 2020-01-29: Annual Review: Background and formatting updated.

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