WHA
 

Gaucher Disease Agents - PA, NF

Indications for Prior Authorization

Cerdelga (eliglustat)
  • For diagnosis of Type 1 Gaucher Disease
    Indicated for the long-term treatment of adult patients with Gaucher disease type 1 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 CYP2D6 indeterminate metabolizers.

Cerezyme (imiglucerase) for injection
  • For diagnosis of Type 1 or Type 3 Gaucher Disease
    Indicated for the treatment of non-central nervous system (CNS) manifestations of Type 1 or Type 3 Gaucher disease in adults and pediatric patients.

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.

Zavesca (miglustat), Yargesa (miglustat), Generic miglustat
  • For diagnosis of Type 1 Gaucher Disease
    Indicated as monotherapy for the treatment of adult patients with mild/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).

  • For diagnosis of Niemann-Pick disease type C (NPC)
    Indicated for use in combination with Miplyffa (arimoclomol) for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older. [12-14]

Criteria

Cerezyme, Elelyso, or VPRIV

Prior Authorization

Length of Approval: 24 Month(s)
For diagnosis of Type 1 Gaucher Disease

  • Diagnosis of Type 1 Gaucher disease
    • AND
  • Patient has evidence of symptomatic disease (e.g., moderate to severe anemia, thrombocytopenia, bone disease, hepatomegaly, or splenomegaly) [A-D, F, G, 17]
    • AND
  • For Elelyso and VPRIV only, patient is 4 years of age or older
Cerezyme

Prior Authorization

Length of Approval: 24 Month(s)
For diagnosis of Type 3 Gaucher Disease

  • Diagnosis of Type 3 Gaucher disease
    • AND
  • Presence of a non-central nervous system (CNS) manifestation (e.g., moderate to severe anemia, thrombocytopenia, bone disease, hepatomegaly, or splenomegaly) [A-D, F, G]
Cerdelga

Prior Authorization

Length of Approval: 24 Month(s)
For diagnosis of Type 1 Gaucher Disease

  • 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 a FDA-approved test or a test performed at a facility approved by Clinical Laboratory Improvement Amendments (CLIA)
    • AND
  • Patient is 18 years of age or older
Brand Zavesca, Generic miglustat, or Yargesa

Prior Authorization

Length of Approval: 24 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

Non Formulary

Length of Approval: 0 OptumRx: 24 months, OptumRx-EHB: 12 months
For diagnosis of Type 1 Gaucher Disease

  • Submission of medical records (e.g., chart notes) confirming diagnosis of mild to moderate Type 1 Gaucher disease
    • AND
  • Patient is 18 years of age or older
    • AND
  • Submission of medical records (e.g., chart notes) or paid claims confirming trial and failure, contraindication, or intolerance to Cerdelga
    • AND
  • Both of the following:
    • Submission of medical records (e.g., chart notes) confirming the patient has experienced intolerance (e.g., allergy to excipient) with Yargesa (miglustat) that has the same active ingredient
    • Submission of medical records confirming Yargesa (miglustat) has not been effective AND valid clinical justification provided explaining how Brand Zavesca is expected to provide benefit when Yargesa (miglustat) has not been shown to be effective despite having the same active ingredient
Brand Zavesca, Generic miglustat, or Yargesa

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, or Yargesa

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)
Brand Zavesca

Non Formulary

Length of Approval: 0 OptumRx: 6 months, OptumRx-EHB: 12 months
For diagnosis of Niemann-Pick disease type C (NPC) (off-label) [E]

  • Submission of medical records (e.g., chart notes) confirming diagnosis of Niemann-Pick disease type C (NPC)
    • AND
  • Submission of medical records (e.g., chart notes) or paid claims confirming requested drug will be used in combination with Miplyffa (arimoclomol)
    • AND
  • Submission of medical records (e.g., chart notes) or paid claims confirming trial and failure, contraindication, or intolerance to Cerdelga
    • AND
  • Both of the following:
    • Submission of medical records (e.g., chart notes) confirming the patient has experienced intolerance (e.g., allergy to excipient) with Yargesa (miglustat) that has the same active ingredient
    • Submission of medical records confirming Yargesa (miglustat) has not been effective AND valid clinical justification provided explaining how Brand Zavesca is expected to provide benefit when Yargesa (miglustat) has not been shown to be effective despite having the same active ingredient
    AND
  • Prescribed by or in consultation with a specialist knowledgeable in the treatment of Niemann-Pick disease type C

  • Guideline ID
    GL-506337

  • Formulary
    Premium

  • Effective date
    Apr 1, 2026

  • P&T approval date
    Nov 20, 2000

P & T Revisions

2026-02-11, 2025-12-31, 2025-03-07, 2025-02-24, 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

  1. Cerezyme Prescribing Information. Genzyme Corporation. Cambridge, MA. January 2026.
  2. Elelyso Prescribing Information. Pfizer, Inc. New York, NY. January 2025.
  3. VPRIV Prescribing Information. Takeda Pharmaceuticals U.S.A., Inc. Lexington, MA. September 2024.
  4. Cerdelga Prescribing Information. Genzyme Corporation. Cambridge, MA. January 2024.
  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. July 2024.
  12. Miplyffa Prescribing Information. Zevra Therapeutics, Inc. Celebration, FL. 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.
  14. FDA Review: Miplyffa. Food and Drug Administration Web Site. 2024. http://www.accessdata.fda.gov. Accessed December 26, 2025.
  15. Miglustat Prescribing Information. ANI Pharmaceuticals, Inc. Baudette, MN. August 2022.
  16. Patterson MC, Hendriksz CJ, Walterfang M, et al; NP-C Guidelines Working Group. Recommendations for the diagnosis and management of Niemann-Pick disease type C: an update. Mol Genet Metabol. 2012; 106(3):330-344.
  17. Hughes D, Sidransky E, Sutton VR, Kremen J. Gaucher disease: Treatment. Wolters Kluwer. Updated June 30, 2025. Available with subscription from https://www.uptodate.com. Accessed December 26, 2025.
  18. Atwal PS, Barron SA, van Zuuren EJ. Gaucher disease. EBSCO. Updated January 16, 2026. Available from: https://www.dynamed.com/condition/gaucher-disease.Accessed January 26, 2026.
  19. El-Beshlawy A, Tylki-Szymanska A, Vellodi A, et al. Long-term hematological, visceral, and growth outcomes in children with Gaucher disease type 3 treated with imiglucerase in the International Collaborative Gaucher Group Gaucher Registry. Mol Genet Metab. 2017; 120(1-2):47-56.
  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. The off-label use of miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in combination with Miplyffa is supported per Miplyffa FDA labelling. [12-14] Additionally, NPC treatment guideline recommends treatment dosage for NPC with neurological manifestations of 200 mg 3 times daily, with neurological status assessed regularly (e.g., every 6 months) and continuation of therapy reevaluated after 1 or more years. [16]
  6. International consensus recommendations on the management of Gaucher disease in children recommend initiating enzyme replacement therapy (ERT) in symptomatic children and adolescents with Type 1 or Type 3 and at least one of the following manifestations: symptomatic disease before 20 years of age, severe anemia, severe thrombocytopenia, leukocytes less than 3000 cells/mL, symptomatic bone disease, active bone disease (including asymptomatic), decreased growth velocity or growth retardation, pubertal delay, sibling with severe disease being managed with ERT, genotype associated with severe disease (e.g., L444P or D409H variants), bone mineral density z-score below -2, or spleen and liver volume greater than 2 times normal. [18, 19]
  7. ERT has not been shown to reduce progression of neurologic symptoms and is therefore not recommended to treat patients with Type 2 Gaucher disease, which is characterized by rapid onset (i.e., first months/years of life) and progression of central nervous system (CNS) involvement leading to infancy fatality and little impact of ERT. In Type 3 Gaucher disease, neurologic involvement still occurs with early onset, but at milder severity and slower progression; marked visceral and hematological disease and cardiac complications occur with Type 3 Gaucher disease. Although ERT is not expected to address the CNS-related manifestations of Type 3 Gaucher disease, ERT was beneficial in addressing the visceral and hematological aspects of the disease (i.e., improvement in hemoglobin, platelet count, liver volume, spleen volume, height Z-score). Type 1 Gaucher disease is characterized by splenomegaly, blood dyscrasias, and orthopedic complications and lack of neurologic involvement. [18, 19]
  • 2026-02-11: Removed age criterion in Cerezyme Type 1 Gaucher disease bucket due to expanded indication and added new criteria for Cerezyme new indication Type 3 Gaucher disease. Updated EHB NF authorization duration to 12 months. Background updates.
  • 2025-12-31: 2026 Annual Review - Added Yargesa to NPC criteria. Updated Zavesca NPC NF criteria to require med records/paid claims confirming use with Miplyffa. Updated Zavesca NF criteria to include ST through formulary alternatives. Updated Cerdelga criteria for standard genetic testing verbiage. Background updates.
  • 2025-03-07: Updated Type 1 Gaucher disease approval duration from 12 months to 24 months.
  • 2025-02-24: 2025 annual review: added NF criteria for Zavesca, background updates.
  • 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.