Agalsidase beta (Fabrazyme) & Pegunigalsidase alfa-iwxj (Elfabrio) (Medicaid Expansion)

Policy ID: ME-I-55-023-06

Section: Injections

Effective Date: January 01, 2022

Revised Date: February 28, 2025

Revision Effective Date: April 01, 2025

Last Reviewed: March 11, 2025

Applies To: Medicaid Expansion Only

Description

Agalsidase beta (Fabrazyme®) and pegunigalsidase alfa-iwxj (Elfabrio®) are an exogenous source of the lysosomal enzyme α-glucosidase A (a-GalA), catalyzing the hydrosis of glycosphinogolipids, including globotriaosylceramide (GL-3), hence reducing its deposition in capillary endothelium of the kidney, heart, brain and other tissue types. α-glucosidase A deficiency, otherwise known as Fabry Disease, is an X- linked genetic disorder of glycosphingolipid metabolism. Deficiency of a-Gal leads to progressive accumulation of glycoshingolipids, predominantly GL-3, in many body tissues, occurring over a period of years. Clinical manifestations of the disease include renal failure, cardiomyopathy and cerebrovascular accidents.

Policy Application

All claims submitted for this policy will be processed according to the policy effective date and associated revision effective dates in effect on the date of service.

Criteria

Coverage is subject to the specific terms of the member's benefit plan.

The use of agalsidase beta (Fabrazyme) may be considered medically necessary when ALL of the following criteria are met:

The prescriber is, or in consult with, a metabolic specialist, geneticist, cardiologist, or specialist in Fabry disease; and
The individual will not be concurrently treated with Galafold (migalastat); and
The individual must have a diagnosis of Fabry disease with the one of the following (as evidenced with submitted documentation):
- In males assigned at birth must meet one of the following (1 or 2):
  - Deficiency of less than 35% of mean normal alpha-galactosidase A (α-Gal A) enzyme activity; or
  - Diagnosis is confirmed to be caused by a pathologic galactosidase alpha gene (GLA); or
- In females assigned at birth all of the following must be met:
  - Diagnosis must be confirmed to be caused by a pathologic galactosidase alpha gene (GLA); and
  - Baseline value for plasma or urinary globotriosylceramide (GL-3) levels greater than or equal to 5 ng/mcL or GL-3 inclusions greater than or equal to 0.3 per kidney interstitial capillary (KIC) as measured in kidney biopsy; and
  - The individual is experiencing one of the following symptoms:
    - Acroparesthesia (burning pain in the extremities); or
    - Impaired glomerular filtration rate (GFR) or proteinuria; or
    - Left ventricular hypertrophy (LVH); or
    - Gastrointestinal manifestations (e.g., diarrhea, abdominal pain); or
    - GL-3 deposits within the kidney.

Initial Authorization: Six (6) months

Reauthorization Criteria

Continuation of therapy with agalsidase beta (Fabrazyme) may be considered medically necessary when the following is met:

The individual must have a decreased urine or plasma Gb3 level or GL-3 inclusions per kidney interstitial capillary (KIC) and experienced improvement in one of the following symptoms since starting treatment with requested product, subject to clinical review:
- Acroparesthesia (burning pain in the extremities); or
- Left ventricular hypertrophy (LVH); or
- Glomerular filtration rate (GFR) and/or proteinuria; or
- Gastrointestinal manifestations (e.g., diarrhea, abdominal pain).

Continuation Authorization: 12 months

The use of agalsidase beta (Fabrazyme) for all other indications not listed in this policy is considered experimental/investigational and therefore non-covered because the safety and/or effectiveness cannot be established by the available published peer-reviewed literature.

Procedure Code

J0180

The use of pegunigalsidase alfa-iwxj (Elfabrio) may be considered medically necessary when ALL of the following criteria are met:

The prescriber is, or in consult with, a metabolic specialist, geneticist, cardiologist, or specialist in Fabry disease; and
The individual will not be concurrently treated with Galafold (migalastat); and
The individual must have a diagnosis of Fabry disease with the one of the following (as evidenced with submitted documentation):
- In males assigned at birth must meet one of the following (1 or 2):
  - Deficiency of less than 35% of mean normal alpha-galactosidase A (α-Gal A) enzyme activity; or
  - Diagnosis is confirmed to be caused by a pathologic galactosidase alpha gene (GLA); or
- In females assigned at birth all of the following must be met:
  - Diagnosis must be confirmed to be caused by a pathologic galactosidase alpha gene (GLA); and
  - Baseline value for plasma or urinary globotriosylceramide (GL-3) levels greater than or equal to 5 ng/mcL or GL-3 inclusions greater than or equal to 0.3 per kidney interstitial capillary (KIC) as measured in kidney biopsy; and
  - The individual is experiencing one of the following symptoms:
    - Acroparesthesia (burning pain in the extremities); or
    - Impaired glomerular filtration rate (GFR) or proteinuria; or
    - Left ventricular hypertrophy (LVH); or
    - Gastrointestinal manifestations (e.g., diarrhea, abdominal pain); or
    - GL-3 deposits within the kidney.

Initial Authorization: Six (6) months

Reauthorization Criteria

Continuation of therapy with pegunigalsidase alfa-iwxj (Elfabrio) may be considered medically necessary when the following is met:

The individual must have a decreased urine or plasma Gb3 level or GL-3 inclusions per kidney interstitial capillary (KIC) and experienced improvement in one of the following symptoms since starting treatment with requested product, subject to clinical review:
- Acroparesthesia (burning pain in the extremities); or
- Left ventricular hypertrophy (LVH); or
- Glomerular filtration rate (GFR) and/or proteinuria; or
- Gastrointestinal manifestations (e.g., diarrhea, abdominal pain).

The use of pegunigalsidase alfa-iwxj (Elfabrio) for all other indications not listed in this policy is considered experimental/investigational and therefore non-covered because the safety and/or effectiveness cannot be established by the available published peer-reviewed literature.

Procedure Code

J2508

NOTE: In addition to the above criteria, product specific dosage and/or frequency limits may apply in accordance with the United States Food and Drug Administration (U.S. FDA)-approved product prescribing information, national compendia, Centers for Medicare and Medicaid Services (CMS) and other peer reviewed resources or evidence-based guidelines.

Diagnosis Code

Covered diagnosis code for J0180 and J2508

E75.21

Professional Statements and Societal Positions Guidelines

Not Applicable

ND Committee Review

Internal Medical Policy Committee 11-23-2021 New policy - Effective January 01, 2022

Adopted Medicaid Expansion specific policy

Internal Medical Policy Committee 11-29-2022 - Effective January 1, 2023

Updated experimental/investigational statement

Internal Medical Policy Committee 11-15-2023 Annual Review - no changes in criteria

Internal Medical Policy Committee 1-16-2024 - Effective March 01, 2024

Updated age for agalsidase beta (Fabrazyme) from eight (8) to two (2)

Internal Medical Policy Committee 3-19-2024 - Effective May 01, 2024

Added pegunigalsidase alfa-iwxj (Elfabrio) to the policy

Internal Medical Policy Committee 7-16-2024 Effective August 01, 2024

Updated criteria based on the DHHS PDL Version 2024.4

Internal Medical Policy Committee 03-11-2025 Effective April 01, 2025

Updated initial and reauthorization criteria required based on the DHHS Version PDL 2025.2

Links

References (PDF)

Disclaimer

Current medical policy is to be used in determining a Member's contract benefits on the date that services are rendered. Contract language, including definitions and specific inclusions/exclusions, as well as state and federal law, must be considered in determining eligibility for coverage. Members must consult their applicable benefit plans or contact a Member Services representative for specific coverage information. Likewise, medical policy, which addresses the issue(s) in any specific case, should be considered before utilizing medical opinion in adjudication. Medical technology is constantly evolving, and the Company reserves the right to review and update medical policy periodically.

Policy ID: ME-I-55-023-05

Section: Injections

Effective Date: January 01, 2022

Revised Date: July 10, 2024

Revision Effective Date: August 01, 2024

Last Reviewed: July 16, 2024

Archived Date: March 31, 2025

Applies To: Medicaid Expansion Only

Description

Policy Application

All claims submitted for this policy will be processed according to the policy effective date and associated revision effective dates in effect on the date of service.

Criteria

Coverage is subject to the specific terms of the member's benefit plan.

The use of agalsidase beta (Fabrazyme) may be considered medically necessary when ALL of the following criteria are met:

The prescriber is, or in consult with, a metabolic specialist, geneticist, cardiologist, or specialist in Fabry disease; and
The individual will not be concurrently treated with Galafold (migalastat); and
The individual must have a diagnosis of Fabry disease with the one of the following (as evidenced with submitted documentation):
- In males assigned at birth:
  - Deficiency of less than 35% of mean normal alpha-galactosidase A (α-Gal A) enzyme activity; and
  - Diagnosis is confirmed to be caused by a pathologic galactosidase alpha gene (GLA); or
- In females assigned at birth and males with a α-Gal A enzyme activity greater than 35 percent:
  - Diagnosis must be confirmed to be caused by a pathologic galactosidase alpha gene (GLA); and
  - Baseline value for plasma or urinary globotriosylceramide (GL-3) levels greater than or equal to 5 ng/mcL or GL-3 inclusions greater than or equal to 0.3 per kidney interstitial capillary (KIC) as measured in kidney biopsy; and
  - The individual is experiencing one of the following symptoms:
    - Acroparesthesia (burning pain in the extremities); or
    - Angiokeratomas (cutaneous vascular lesions); or
    - Hypo- or anhidrosis (diminished perspiration); or
    - Corneal and lenticular opacities; or
    - Left ventricular hypertrophy (LVH), hypertrophic cardiomyopathy, or arrythmia of unknown etiology; or
    - Chronic kidney disease (CKD), multiple renal cysts, and/or proteinuria of unknown etiology.

Initial Authorization: Six (6) months

Reauthorization Criteria

Continuation of therapy with agalsidase beta (Fabrazyme) may be considered medically necessary when the following is met:

The individual must have a decreased Gb3 level or Cb3 inclusion per KIC level and experienced and maintained improvement in one of the following symptoms since starting treatment with requested product, as evidenced by medical documentation (e.g., chart notes) attached to the request (subject to clinical review):
- Acroparesthesia (burning pain in the extremities); or
- Angiokeratomas (cutaneous vascular lesions); or
- Hypo- or anhidrosis (diminished perspiration); or
- Corneal and lenticular opacities; or
- Left ventricular hypertrophy (LVH), hypertrophic cardiomyopathy, or arrythmia of unknown etiology; or
- Chronic kidney disease (CKD), multiple renal cysts, and/or proteinuria of unknown etiology.

Continuation Authorization: 12 months

Procedure Code

J0180

The use of pegunigalsidase alfa-iwxj (Elfabrio) may be considered medically necessary when ALL of the following criteria are met:

The prescriber is, or in consult with, a metabolic specialist, geneticist, cardiologist, or specialist in Fabry disease; and
The individual will not be concurrently treated with Galafold (migalastat); and
The individual must have a diagnosis of Fabry disease with the one of the following (as evidenced with submitted documentation):
- In males assigned at birth:
  - Deficiency of less than 35% of mean normal alpha-galactosidase A (α-Gal A) enzyme activity; and
  - Diagnosis is confirmed to be caused by a pathologic galactosidase alpha gene (GLA); or
- In females assigned at birth and males with a α-Gal A enzyme activity greater than 35 percent:
  - Diagnosis must be confirmed to be caused by a pathologic galactosidase alpha gene (GLA); and
  - Baseline value for plasma or urinary globotriosylceramide (GL-3) levels greater than or equal to 5 ng/mcL or GL-3 inclusions greater than or equal to 0.3 per kidney interstitial capillary (KIC) as measured in kidney biopsy; and
  - The individual is experiencing one of the following symptoms:
    - Acroparesthesia (burning pain in the extremities); or
    - Angiokeratomas (cutaneous vascular lesions); or
    - Hypo- or anhidrosis (diminished perspiration); or
    - Corneal and lenticular opacities; or
    - Left ventricular hypertrophy (LVH), hypertrophic cardiomyopathy, or arrythmia of unknown etiology; or
    - Chronic kidney disease (CKD), multiple renal cysts, and/or proteinuria of unknown etiology; and
The individual must have failed a trial of agalsidase beta (Fabrazyme), as evidenced by paid claims; or
An explanation has been provided with the request why agalsidase beta (Fabrazyme) cannot be used.

Initial Authorization: Six (6) months

Reauthorization Criteria

Continuation of therapy with pegunigalsidase alfa-iwxj (Elfabrio) may be considered medically necessary when the following is met:

The individual must have a decreased Gb3 level or Cb3 inclusion per KIC level and experienced and maintained improvement in one of the following symptoms since starting treatment with requested product, as evidenced by medical documentation (e.g., chart notes) attached to the request (subject to clinical review):
- Acroparesthesia (burning pain in the extremities); or
- Angiokeratomas (cutaneous vascular lesions); or
- Hypo- or anhidrosis (diminished perspiration); or
- Corneal and lenticular opacities; or
- Left ventricular hypertrophy (LVH), hypertrophic cardiomyopathy, or arrythmia of unknown etiology; or
- Chronic kidney disease (CKD), multiple renal cysts, and/or proteinuria of unknown etiology.

Procedure Code

J2508

Diagnosis Code

Covered diagnosis code for J0180 and J2508

E75.21

Professional Statements and Societal Positions Guidelines

Not Applicable

ND Committee Review

Internal Medical Policy Committee 11-23-2021 New policy - Effective January 01, 2022

Adopted Medicaid Expansion specific policy

Internal Medical Policy Committee 11-29-2022 - Effective January 1, 2023

Updated experimental/investigational statement

Internal Medical Policy Committee 11-15-2023 Annual Review - no changes in criteria

Internal Medical Policy Committee 1-16-2024 - Effective March 01, 2024

Updated age for agalsidase beta (Fabrazyme) from eight (8) to two (2)

Internal Medical Policy Committee 3-19-2024 - Effective May 01, 2024

Added pegunigalsidase alfa-iwxj (Elfabrio) to the policy

Internal Medical Policy Committee 7-16-2024 Effective August 01, 2024

Updated criteria based on the DHHS PDL Version 2024.4

Medical Policies Quick Search

Agalsidase beta (Fabrazyme) & Pegunigalsidase alfa-iwxj (Elfabrio) (Medicaid Expansion)

Description

Policy Application

Criteria

Procedure Code

Procedure Code

Diagnosis Code

Covered diagnosis code for J0180 and J2508

Professional Statements and Societal Positions Guidelines

ND Committee Review

Links

Disclaimer

Description

Policy Application

Criteria

Procedure Code

Procedure Code

Diagnosis Code

Covered diagnosis code for J0180 and J2508

Professional Statements and Societal Positions Guidelines

ND Committee Review

Links

Disclaimer