Idursulfase (Elaprase) (Medicaid Expansion)

Policy ID: ME-I-93-019-04

Section: Injections

Effective Date: January 01, 2022

Revised Date: March 09, 2025

Revision Effective Date: May 01, 2025

Applies To: Medicaid Expansion Only

Description

Idursulfase (Elaprase®), is a purified form of human iduronate-2-sulfatase used for the treatment of Hunter syndrome (Mucopolysaccharidosis II, MPS II) a rare, genetic disease which can lead to premature death. Hunter syndrome is characterized by glycosaminoglycan accumulation due to deficiency in the enzyme iduronate 2-sulfase, which is needed to adequately break down complex sugars produced in the body. Hunter syndrome usually becomes apparent in children one (1) to three (3) years of age. Symptoms include cognitive deterioration, growth delay, joint stiffness, and coarsening of facial features. In severe cases, individuals experience respiratory and cardiac problems, neurological deficits, and death. Individuals with the syndrome do not produce enough of a functional lysosomal enzyme called iduronate-2-sulfatase (IDS) because of this genetic defect.

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 idursulfase (Elaprase) may be considered medically necessary when ALL of the following criteria are met:

The individual must meet FDA-approved label for use (e.g., use outside of studied population will be considered investigational); and
Documentation of the member's diagnosis must be submitted, as evidenced by the following:
- Deficiency in iduronate-2 sulfatase (I2S) enzyme activity of less than or equal to 10% of the lower limit of normal in white cells, fibroblasts, or plasma in the presence of normal activity of at least one other sulfatase; or
- Genetic testing confirming pathogenic mutations in the IDS gene that leaves the Fragile X Messenger Ribonucleoprotein genes ( FMR1 , FMR2 ) intact; and
Idursulfase (Elaprase) must be prescribed by, or in consult with, a geneticist, metabolic specialist, or specialist in mucopolysaccharidoses (MPS); and
The individual does not have severe cognitive or neurologic impairment (e.g., inability to swallow); and
Documentation of one of the following must be submitted:
- The Forced Vital Capacity (FVC) via Pulmonary Function Test; or
- Urinary glycosaminoglycan (uGAG) levels are elevated defined by laboratory reference range; or
- Six (6) -minute walk test (6MWT); or
- Hepatomegaly (liver size 1.25 or more times normal); or
- Splenomegaly (spleen size five (5) or more times normal).

Reauthorization Criteria

Continuation of therapy with idursulfase (Elaprase) may be considered medically necessary when ALL of the following are met:

Documentation must be submitted confirming improvement of one of the following:
- The Forced Vital Capacity (FVC) via Pulmonary Function Test relative improvement of 10% over baseline; or
- Urinary glycosaminoglycan (uGAG) levels normalization defined by laboratory reference range; or
- Six (6) -minute walk test (6MWT) increase; or
- Reduction in liver volume to normal size or by 10%; or
- Reduction in spleen volume by 15%.

The use of idursulfase (Elaprase) 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

J1743

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

E76.1

Professional Statements and Societal Positions Guidelines

Not Applicable

ND Committee Review

Internal Medical Policy Committee 11-23-2021- Effective January 1, 2022

Adopted Medicaid Expansion specific policy

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

Updated initial criteria, and
Updated reauthorization criteria, and
Updated experimental/investigational statement

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

Internal Medical Policy Committee 11-19-2024 Effective December 01, 2024

Updated criteria to remove ' The individual's age must be five (5) years of age or older' based on the DHHS PDL Version 2024.6

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

Updated initial criteria required based on the DHHS PDL Version 2025.3

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-93-019-03

Section: Injections

Effective Date: January 01, 2022

Revised Date: October 10, 2024

Revision Effective Date: December 01, 2024

Last Reviewed: November 19, 2024

Archived Date: April 30, 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 idursulfase (Elaprase) may be considered medically necessary when ALL of the following criteria are met:

The individual must meet FDA-approved label for use (e.g., use outside of studied population will be considered investigational); and
Documentation of the member's diagnosis must be submitted, as evidenced by the following:
- Deficiency in iduronate-2 sulfatase (I2S) enzyme activity in white cells, fibroblasts, or plasma in the presence of normal activity of at least one other sulfatase; or
- Genetic testing confirming pathogenic mutations in the IDS gene; and
Idursulfase (Elaprase) must be prescribed by, or in consult with, an expert in lysosomal storage diseases; and
The individual does not have severe cognitive or neurologic impairment (e.g., inability to swallow); and
Documentation of one of the following must be submitted:
- The Forced Vital Capacity (FVC) via Pulmonary Function Test; or
- Urinary glycosaminoglycan (uGAG) levels are elevated defined by laboratory reference range; or
- Six (6) -minute walk test (6MWT); or
- Hepatomegaly (liver size 1.25 or more times normal); or
- Splenomegaly (spleen size five (5) or more times normal).

Reauthorization Criteria

Continuation of therapy with idursulfase (Elaprase) may be considered medically necessary when ALL of the following are met:

Documentation must be submitted confirming improvement of one of the following:
- The Forced Vital Capacity (FVC) via Pulmonary Function Test relative improvement of 10% over baseline; or
- Urinary glycosaminoglycan (uGAG) levels normalization defined by laboratory reference range; or
- Six (6) -minute walk test (6MWT) increase; or
- Reduction in liver volume to normal size or by 10%; or
- Reduction in spleen volume by 15%.

Procedure Code

J1743

Diagnosis Code

E76.1

Professional Statements and Societal Positions Guidelines

Not Applicable

ND Committee Review

Internal Medical Policy Committee 11-23-2021- Effective January 1, 2022

Adopted Medicaid Expansion specific policy

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

Updated initial criteria, and
Updated reauthorization criteria, and
Updated experimental/investigational statement

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

Internal Medical Policy Committee 11-19-2024 Effective December 01, 2024

Updated criteria to remove ' The individual's age must be five (5) years of age or older' based on the DHHS PDL Version 2024.6

Medical Policies Quick Search

Idursulfase (Elaprase) (Medicaid Expansion)

Description

Policy Application

Criteria

Procedure Code

Diagnosis Code

Professional Statements and Societal Positions Guidelines

ND Committee Review

Links

Disclaimer

Description

Policy Application

Criteria

Procedure Code

Diagnosis Code

Professional Statements and Societal Positions Guidelines

ND Committee Review

Links

Disclaimer