Highmark Commercial Medical Policy - Pennsylvania

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Medical Policy: I-55-017
Topic: Agalsidase beta (Fabrazyme)
Section: Injections
Effective Date: May 28, 2018
Issue Date: May 28, 2018
Last Reviewed: February 2018

Agalsidase beta (Fabrazyme®) serves as 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.

This policy is designed to address medical guidelines that are appropriate for the majority of individuals with a particular disease, illness, or condition. Each person's unique clinical circumstances may warrant individual consideration, based on review of applicable medical records.

Policy Position Coverage is subject to the specific terms of the member’s benefit plan.

Aglasidas beta (Fabrazyme) may be considered medically necessary for the treatment of Fabry disease when ALL of the following criteria are met:

  • Individuals eight (8) years of age or older with a confirmed diagnosis of Fabry disease made by ONE of the following methods:
    • For male individuals: clinical documentation of complete deficiency or negligible (less than 5%) of mean normal alpha-galactosidase A (α-Gal A) enzyme activity in leukocytes, dried blood spots, or serum (plasma) analysis; or
    • For female individuals: documented galactosidase alpha gene; (GLA) mutation by gene sequencing; and
  • ONE or more of the following symptoms or physical findings attributable to Fabry disease:
    • Angiokeratomas; (clusters of small, dark red spots on the skin); or
    • Acroparesthesias; (episodes of pain, particularly in the hands and feet); or
    • Corneal verticillata (whorls); or
    • Corneal opacity; or  
    • Personal or family history of exercise, heat, or cold intolerance; or
    • Decreased sweating (anhidrosis or hypohidrosis); or
    • Personal or family history of renal failure; or.
    • Anhydrosis or hypohidrosis (impaired sweating); or
    • Hearing manifestations (tinnitus); and
  • The medication is prescribed by or in consultation with a physician who specializes in the treatment of inherited metabolic disorders.

Aglasidas beta (Fabrazyme) for any other indication is considered experimental/investigational, and therefore, non-covered.

Procedure Codes

Agalsidase beta (Fabrazyme) is not reimbursable under the prescription drug benefit.

NOTE: Dosage recommendations per the FDA label.

Place of Service: Outpatient

Experimental/Investigational (E/I) services are not covered regardless of place of service.

The policy position applies to all commercial lines of business

Denial Statements

Services that do not meet the criteria of this policy will be considered experimental/investigational (E/I). A network provider can bill the member for the experimental/investigational service. The provider must give advance written notice informing the member that the service has been deemed E/I. The member must be provided with an estimate of the cost and the member must agree in writing to assume financial responsibility in advance of receiving the service. The signed agreement must be maintained in the provider’s records.


Medical policies do not constitute medical advice, nor are they intended to govern the practice of medicine. They are intended to reflect Highmark's reimbursement and coverage guidelines. Coverage for services may vary for individual members, based on the terms of the benefit contract.

Discrimination is Against the Law
The Claims Administrator/Insurer complies with applicable Federal civil rights laws and does not discriminate on the basis of race, color, national origin, age, disability, or sex. The Claims Administrator/Insurer does not exclude people or treat them differently because of race, color, national origin, age, disability, or sex. The Claims Administrator/ Insurer:
  • Provides free aids and services to people with disabilities to communicate effectively with us, such as:
    • Qualified sign language interpreters
    • Written information in other formats (large print, audio, accessible electronic formats, other formats)
  • Provides free language services to people whose primary language is not English, such as:
    • Qualified interpreters
    • Information written in other languages
If you need these services, contact the Civil Rights Coordinator.

If you believe that the Claims Administrator/Insurer has failed to provide these services or discriminated in another way on the basis of race, color, national origin, age, disability, or sex, you can file a grievance with: Civil Rights Coordinator, P.O. Box 22492, Pittsburgh, PA 15222, Phone: 1-866-286-8295, TTY: 711, Fax: 412-544-2475, email: CivilRightsCoordinator@highmarkhealth.org. You can file a grievance in person or by mail, fax, or email. If you need help filing a grievance, the Civil Rights Coordinator is available to help you.

You can also file a civil rights complaint with the U.S. Department of Health and Human Services, Office for Civil Rights electronically through the Office for Civil Rights Complaint Portal, available at https://ocrportal.hhs.gov/ocr/portal/lobby.jsf, or by mail or phone at:

U.S. Department of Health and Human Services
200 Independence Avenue, SW
Room 509F, HHH Building
Washington, D.C. 20201
1-800-368-1019, 800-537-7697 (TDD)

Complaint forms are available at http://www.hhs.gov/ocr/office/file/index.html.

Insurance or benefit/claims administration may be provided by Highmark, Highmark Choice Company, Highmark Coverage Advantage, Highmark Health Insurance Company, First Priority Life Insurance Company, First Priority Health, Highmark Benefits Group, Highmark Select Resources, Highmark Senior Solutions Company or Highmark Senior Health Company, all of which are independent licensees of the Blue Cross and Blue Shield Association, an association of independent Blue Cross and Blue Shield plans.

Highmark retains the right to review and update its medical policy guidelines at its sole discretion. These guidelines are the proprietary information of Highmark. Any sale, copying or dissemination of the medical policies is prohibited; however, limited copying of medical policies is permitted for individual use.

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