CurrentCareSourcePolicy N/A

Enzyme Replacement Therapy — Elaprase (idursulfase) Coverage Criteria

This policy governs prior authorization and medical necessity criteria for Elaprase (idursulfase) intravenous infusion for treatment of Mucopolysaccharidosis type II (Hunter syndrome) for CareSource members.

Policy Summary

PayerCareSource

PolicyEnzyme Replacement Therapy — Elaprase (idursulfase) Coverage Criteria

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateN/A

Next Review DateApr 16, 2025

Key ActionObtain prior authorization and document diagnostic confirmation and specialist involvement; approve for 1 year if all criteria and dosing (≤0.5 mg/kg IV no more frequently than weekly) are met.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

1 yrapproval duration

0.5 mg/kgmax dose

weeklyfrequency

NCapplicable state

Recommended Authorization Criteria

inv-01: COVERAGE CRITERIA

Covered when ALL of the following are met

FDA-Approved Indication

Diagnostic confirmation: Diagnosis established by ONE of: (i) laboratory test demonstrating deficient iduronate-2-sulfatase activity in leukocytes, fibroblasts, serum, or plasma; OR (ii) molecular genetic test demonstrating an iduronate-2-sulfatase gene variant
Prescriber requirement: Elaprase is prescribed by or in consultation with a geneticist, endocrinologist, metabolic disorder subspecialist, or physician who specializes in lysosomal storage disorders
Dosing requirement: Each dose must not exceed 0.5 mg/kg administered intravenously and doses are given no more frequently than once weekly<=0.5 mg/kg weekly

Policy Summary

PayerCareSource

PolicyEnzyme Replacement Therapy — Elaprase (idursulfase) Coverage Criteria

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateN/A

Next Review DateApr 16, 2025

SourceLink

On This Page

Trek Health ingests and normalizes Transparency in Coverage data and payer policy updates to give provider organizations a clear view of how commercial reimbursement behaves across markets, payers, and services. Our platform transforms raw payer disclosures into structured intelligence that supports contract evaluation, payer negotiations, and service line strategy. By combining market benchmarks with ongoing policy visibility, Trek helps teams identify variability, risk, and opportunity in commercial reimbursement. The result is faster insight, stronger negotiating positions, and more informed financial decisions.

Prior Authorization, Documentation, and Review

Prior Authorization

Prior Authorization Recommended

Prior Authorization is recommended for medical benefit coverage of Elaprase. Approvals are recommended for 1 year when all criteria are met and dosing follows the policy (each dose ≤ 0.5 mg/kg IV no more frequently than once weekly). All approvals are provided for the duration noted above. Automation: None.

Approve for 1 year if ALL criteria are met and dosing follows policy
Requests for doses outside established dosing will be reviewed case-by-case by a clinician (Medical Director or Pharmacist)

Documentation Required

Dose Exception Review

Requests for doses outside the established dosing (each dose must not exceed 0.5 mg/kg IV no more frequently than once weekly) will be considered on a case-by-case basis. A clinician (Medical Director or Pharmacist) will review dose-exception requests and may approve when medically justified documentation is provided.

Dose exception requests require clinician review (Medical Director or Pharmacist)
Provide clinical rationale and supporting documentation when requesting doses outside policy limits

Documentation Required

Required Diagnostic Documentation and Specialist Involvement

Providers must document diagnostic confirmation and specialist involvement as follows: 1) Diagnosis of Mucopolysaccharidosis Type II (Hunter syndrome) confirmed by ONE of the following: a) laboratory testing demonstrating deficient iduronate-2-sulfatase activity in leukocytes, fibroblasts, serum, or plasma; OR b) molecular genetic testing demonstrating a pathogenic/iduronate-2-sulfatase gene variant. 2) Elaprase must be prescribed by, or in consultation with, a specialist experienced in lysosomal storage disorders — examples include a geneticist, endocrinologist, metabolic disorder sub-specialist, or a physician who specializes in the treatment of lysosomal storage disorders. Include copies of laboratory or genetic reports and documentation of specialist involvement with the authorization request.

Required diagnostic documentation: deficient iduronate-2-sulfatase activity OR pathogenic variant on molecular genetic testing
Specialist involvement: prescription by or documentation of consultation with a geneticist, endocrinologist, metabolic disorder subspecialist, or lysosomal storage disorder specialist
Attach laboratory/genetic reports and consult notes with the PA request

Denial Risk

Non‑listed Indications

Coverage is not recommended for indications or circumstances not listed in the Recommended Authorization Criteria. Requests outside the documented criteria will be reviewed separately and are generally not recommended for approval.

Not recommended for unlisted indications; criteria will be updated as new published data become available

Clinical Background

Mucopolysaccharidosis type II (Hunter syndrome) is an X-linked lysosomal storage disorder caused by deficiency of iduronate-2-sulfatase, resulting in accumulation of glycosaminoglycans such as dermatan sulfate and heparan sulfate. The disease predominantly affects males and presents with variable onset and severity, including neurologic involvement in severe forms and multisystem manifestations (coarse facial features, hepatosplenomegaly, cardiac and respiratory disease, short stature, and joint contractures). Enzyme replacement therapy with Elaprase (idursulfase) is used to replace the deficient enzyme and reduce glycosaminoglycan accumulation; definitive diagnosis is by demonstration of deficient enzyme activity or by identification of a pathogenic variant in the iduronate-2-sulfatase gene.

OpenPayer

Enzyme Replacement Therapy — Elaprase (idursulfase) Coverage Criteria

Recommended Authorization Criteria

Dose Limits and Coding Notes

Prior Authorization, Documentation, and Review

Clinical Background

Definitive Diagnosis