Elfabrio®

Drug - Elfabrio® (pegunigalsidase alfa-iwx injection) [Chiesi USA, Inc.]

March 2025

Therapeutic Area - Fabry Disease

Initial approval criteria

Patient is at least 18 years of age; AND

Universal Criteria

Fabry Disease (alpha-galactosidase A deficiency)

Documented diagnosis of Fabry disease with biochemical/genetic confirmation by one of the following:
- α-galactosidase A (α-Gal A) activity in plasma, isolated leukocytes, and/or cultured cells (males only); OR
- Detection of pathogenic mutations in the GLA gene by molecular genetic testing; AND
Baseline value for plasma GL-3 and/or GL-3 inclusions, plasma or urinary globotriaosylceramide (Gb3/GL-3); or plasma globotriaosylsphingosine (lyso-Gb3)

Patient continues to meet universal and other indication-specific relevant criteria such as concomitant therapy requirements (not including prerequisite therapy), performance status, etc. identified in the initial approval criteria; AND
Absence of unacceptable toxicity from the drug. Examples of unacceptable toxicity include: anaphylaxis and severe hypersensitivity reactions, severe infusion-associated reactions, glomerulonephritis, etc.; AND
Disease response with treatment as defined by a reduction or stabilization in one or more of the following, as compared to pre-treatment baseline:
- plasma GL-3 and/or GL-3 inclusions
- plasma or urinary globotriaosylceramide (Gb3/GL-3)
- plasma globotriaosylsphingosine (lyso-Gb3); OR
Disease response with treatment as defined by an improvement or a stabilization in the rate of decline of the estimated glomerular filtration rate (eGFR)

1 mg/kg of actual body weight infused every two weeks as an intravenous (IV) infusion
Patient’s actual body weight must be submitted at the time of request

Elfabrio must be billed as a medical claim.

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