Agents to treat Fabry's Disease: ELFABRIO (pegunigalsidase alfa-iwxi), FABRAZYME (agalsidease beta), GALAFOLD (migalastat)

Agents to treat Fabry's Disease: ELFABRIO (pegunigalsidase alfa-iwxi), FABRAZYME (agalsidease beta), GALAFOLD (migalastat)

Elfabrio, Fabrazyme: Medical Administration – intravenous

Galafold: Self-Administration – oral 

Diagnosis considered for coverage:

Fabry Disease- indicated for the treatment of adults with confirmed Fabry disease. 

Coverage Criteria:

For diagnosis of Fabry disease

• Diagnosis of Fabry disease, AND
• One of the following:
  • Elfabrio or Galafold: Patient is 18 years of age or older, or
  • Fabrazyme: Patient is 2 years of age or older, AND
• Disease confirmed by one of the following:
  • Detection of pathogenic mutations in the GLA gene by molecular genetic testing, or
  • Deficiency in α-galactosidase A (α-Gal A) enzyme activity in plasma (<1.5 nmol/hr/ml in plasma), isolated leukocytes (<4 nmol/hrmg in leukocytes) or dried blood spots (DBS), or 
  • Significant clinical manifestations (e.g., neuropathic pain, cardiomyopathy, renal insufficiency, angiokeratomas, cornea verticillate), AND
• Patient has an amenable galactosidase alpha gene (GLA) variant based on in vitro assay date (Galafold only), AND
• One of the following:
  • Elfabrio or Fabrazyme: Will not be used in combination with Galafold, or
  • Galafold: Will not be used in combination with Fabrazyme

Reauthorization Criteria:

For diagnosis of Fabry disease:

• Documentation of positive clinical response to therapy (e.g. reduction in plasma or urinary sediment lyso-GL-3, GL-3 compared to baseline, reduction in number of GL- inclusions per kidney interstitial capillary (KIC) in renal biopsy samples compared to baseline, improvement and/or stabilization in symptoms (e.g. renal function, neuropathic pain)), AND
• One of the following:
  • Elfabrio or Fabrazyme: Will not be used in combination with Galafold, or
  • Galafold: Will not be used in combination with Fabrazyme
• For Fabrazyme only: duration of treatment does not exceed 60 months

Coverage Duration: 

Elfabrio (pegunigalsidase alfa-iwxj)
Initial: 12 months
Reauthorization: 24 months

Fabrazyme (agalsidase beta)
Initial: 12 months
Reauthorization: 24 months
Maximum of 60 months duration of treatment

Galafold (migalastat)
Initial: 12 months
Reauthorization: 24 months

Dosing: 

Elfabrio and Fabrazyme: 1 mg/kg IV infusion every 2 weeks
Galafold only: Recommended dose is 123 mg orally once every other day (QOD). Take Galafold on an empty stomach, do not consume food at least 2 hours after taking Galafold.

Authorization is not covered for the following:

The use of this drug for indications not listed in this policy does not meet the coverage criteria established by the Western Health Advantage (WHA) Pharmacy and Therapeutics (P&T) Committee.

Policy Updates:

• 12/01/2014 – New policy approved by P&T for Fabrazyme.
• 05/31/2019 – New policy approved by P&T for Galafold.
• 12/01/2023 (policy effective date)- Treatment for Fabry’s Disease Criteria update, including Elfabrio to the list (P&T 11/14/2023) (P&T meeting November) 

References:

1. Elfabrio. IBM Micromedex Solutions. Truven Health Analytics, Inc. Ann Arbor, MI. Accessed September 21, 2023. http://www.micromedexsolutions.com.
2. Fabrazyme. IBM Micromedex Solutions. Truven Health Analytics, Inc. Ann Arbor, MI. Accessed September 26, 2023. http://www.micromedexsolutions.com.
3. Fabrazyme prescribing information. Genzyme Corporation. Cambridge, MA. August 2021. 
4. 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. 
5. 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. 
6. Elfabrio prescribing information. Chiesi USA, Inc. Cary, NC. May 2023. 
7. Galafold prescribing information. Amicus Therapeutics U.S., Inc. Cranbury, NJ. February 2021. 
8. 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. 
9. 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. 
10. Galafold. IBM Micromedex Solutions. Truven Health Analytics, Inc. Ann Arbor, MI. Accessed October 4, 2023. http://www.micromedexsolutions.com.