Pharmacy Prior Authorization Criteria

Fabry Disease Agents

Indications for Prior Authorization

Fabrazyme (agalsidase beta)

• For diagnosis of Fabry disease
  Indicated for the treatment of adult and pediatric patients 2 years of age and older with confirmed Fabry disease.

Elfabrio (pegunigalsidase alfa-iwxj)

• For diagnosis of Fabry disease
  Indicated for the treatment of adults with confirmed Fabry disease.

Criteria

Fabrazyme

Prior Authorization (Initial Authorization)

Length of Approval: 12 Month(s)

• Diagnosis of Fabry disease
AND
• Patient is 2 years of age or older
AND
• One of the following: [3, 4]
• Detection of pathogenic mutations in the GLA gene by molecular genetic testing
• Deficiency in α-galactosidase A (α-Gal A) enzyme activity in plasma, isolated leukocytes, or dried blood spots (DBS)
• Significant clinical manifestations (e.g., neuropathic pain, cardiomyopathy, renal insufficiency, angiokeratomas, cornea verticillata)
AND
• Will not be used in combination with other drugs used for Fabry disease [A]

Fabrazyme

Prior Authorization (Reauthorization)

Length of Approval: 24 Month(s)

• Patient demonstrates positive clinical response to therapy

Elfabrio

Prior Authorization (Initial Authorization)

Length of Approval: 12 Month(s)

• Diagnosis of Fabry disease
AND
• Disease confirmed by one of the following: [3, 4]
• Detection of pathogenic mutations in the GLA gene by molecular genetic testing
• Deficiency in α-galactosidase A (α-Gal A) enzyme activity in plasma, isolated leukocytes, or dried blood spots (DBS)
• Significant clinical manifestations (e.g., neuropathic pain, cardiomyopathy, renal insufficiency, angiokeratomas, cornea verticillata)
AND
• Will not be used in combination with other drugs used for Fabry Disease [A]

Elfabrio

Prior Authorization (Reauthorization)

Length of Approval: 24 Month(s)

• Patient demonstrates positive clinical response to therapy

P & T Revisions

2024-10-02, 2024-07-31, 2023-12-12, 2023-11-01, 2023-08-21, 2023-06-29, 2022-09-30, 2021-10-21, 2021-09-27, 2021-05-12, 2020-10-02, 2019-09-19

References

1. Fabrazyme prescribing information. Genzyme Corporation. Cambridge, MA. July 2024.
2. Per clinical consultation with geneticist. October 11, 2018.
3. Ortiz A, Germain DP, Desnick RJ, et al. Fabry disease revisited: Management and treatment recommendations for adult patients. Mol Genet Metab. 2018;123(4):416-427. doi:10.1016/j.ymgme.2018.02.014.
4. Michaud M, Mauhin W, Belmatoug N, et al. When and How to Diagnose Fabry Disease in Clinical Pratice. Am J Med Sci. 2020;360(6):641-649. doi:10.1016/j.amjms.2020.07.011.
5. Elfabrio prescribing information. Chiesi USA, Inc. Cary, NC. May 2023.
6. UptoDate. Fabry disease:Treatment and prognosis. Available at: https://www.uptodate.com/contents/fabry-disease-treatment-and-prognosis?search=fabry%20disease&source=search_result&selectedTitle=2~68&usage_type=default&display_rank=2. Accessed September 16, 2024.

---

End Notes

1. The safety and effectiveness of concomitant use of Galafold (migalastat) and Fabrazyme (agalsidase beta) has not been established. [2, 6]

---

Revision History

• 2024-10-02: 2024 Annual Review. No criteria changes. Updated references.
• 2024-07-31: Added Elfabrio 5mg/2.5ml IV solution to guideline.
• 2023-12-12: Updated reauth verbiage
• 2023-11-01: Annual Review, no changes.
• 2023-08-21: update guideline
• 2023-06-29: New UM PA Criteria for Elfabrio
• 2022-09-30: 2022 UM Annual Review.
• 2021-10-21: Background updates.
• 2021-09-27: Background updates.
• 2021-05-12: Background updates.
• 2020-10-02: Removed drug name from criteria.
• 2019-09-19: Annual Review - no change to clinical criteria.

---