CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Elaprase (idursulfase) (Intravenous) — Coverage Criteria for Mucopolysaccharidosis II (Hunter syndrome)

This policy governs coverage, dosing limits, authorization length, and clinical criteria for Elaprase (idursulfase) intravenous enzyme replacement therapy for members with Mucopolysaccharidosis II (Hunter syndrome). It applies to clinical reviewers, prescribing providers, and pharmacy/infusion services handling coverage decisions.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyElaprase (idursulfase) (Intravenous) — Coverage Criteria for MPS II

Policy CodePolicy N/A

Change TypeNo material change

Effective DateMar 1, 2020

Next Review DateJan 10, 2024

Key ActionObtain prior authorization for 12 months and document definitive diagnosis plus baseline measures to enable approval and renewal.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

12 moLength of authorization

240 u/28dDosing limit

0.5 mg/kgDose administered

J1743HCPCS code

E76.1Covered ICD-10

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Coverage Criteria for Elaprase (idursulfase)

Initial Therapy — Covered when ALL of the following are met.

Covered when ALL of the following are met:

Initial approval

Baseline values (select one or more by age)
- Patients >= 5 years: One or more of: 6-minute walk test (6-MWT), percent predicted forced vital capacity (FVC), joint range of motion, left ventricular hypertrophy, growth, quality of life (CHAQ/HAQ/MPS HAQ), and/or urinary glycosaminoglycan (uGAG).
- Patients 16 months to <5 years: One or more of: spleen volume, liver volume, FVC, 6-minute walk test (6-MWT), and/or urinary glycosaminoglycan (uGAG).

Continuation/Renewal — Renewal covered when ALL of the following are met.

Renewal covered when ALL of the following are met:

Renewal: Patient continues to meet indication-specific relevant criteria; AND there is absence of unacceptable toxicity from the drug (examples include severe hypersensitivity reactions including anaphylaxis, antibody development with serious adverse reactions, acute respiratory complications, acute cardiorespiratory failure); AND patient has demonstrated a beneficial response to therapy compared to pretreatment age-appropriate baseline in one or more specified measures (stabilization or improvement in percent predicted FVC or 6-MWT, increased joint range of motion, decreased left ventricular hypertrophy, improved growth, improved quality of life, and/or uGAG levels for patients >=5 years; or reductions in spleen and/or liver volume, or stabilization/improvement in FVC and/or 6-MWT, or uGAG levels for patients 16 months to <5 years).clinical improvement or stabilization vs baseline

This therapy is intended to treat the non‑central nervous system (non‑CNS) manifestations of Mucopolysaccharidosis II (Hunter syndrome). Coverage requires that the patient does not have severe, irreversible cognitive impairment and that treatment is directed at somatic features (for example, mobility, pulmonary function, organomegaly, growth, joint range of motion, and urinary glycosaminoglycan levels) rather than primary CNS disease.

For continuation/renewal, coverage may be denied when there is unacceptable toxicity from the drug (examples include severe hypersensitivity reactions such as anaphylaxis, antibody development with serious adverse reactions, acute respiratory or cardiorespiratory complications) or when the patient has shown no demonstrated benefit compared with pretreatment age‑appropriate baseline measures.

Molecular genetic testing of IDS gene — pathogenic mutation satisfies diagnostic confirmation requirement for coverage.

Diagnostic confirmation by molecular testing: Detection of a pathogenic mutation in the IDS gene by molecular genetic testing satisfies the diagnostic confirmation requirement for MPS II.

Eligibility requirements for prior authorization and coverage are specified in the Initial Approval and Renewal criteria sections and include age, diagnostic confirmation, baseline documentation, and indication‑specific clinical measures. See the coverage criteria for the full list of required elements.

Coding and Dosing Limits

Covered HCPCS CodesHCPCSCovered

J1743

Injection, idursulfase, 1 mg; 1 mg = 1 billable unit

NDCNDCCovered

54092-0700-xx

Elaprase 6 mg/3 mL single-use vial for injection

Covered ICD-10 CodesICD-10Covered

E76.1

Mucopolysaccharidosis, type II

HCPCS dosing limits

HCPCS unit dosing limitMaximum 240 billable units every 28 days

Billing intervalPer 28-day (4-week) period

Unit definitionHCPCS units correspond to J1743 (1 mg idursulfase = 1 billable unit)

Provider Actions, Authorization, and Documentation

Prior Authorization

Prior Authorization Required

Prior authorization required. Coverage is provided for 12 months and may be renewed when renewal criteria and documentation are met.

Step Therapy

Step Therapy Exception for Medicare

Medicare members who have previously received this medication within the past 365 days are exempt from Step Therapy requirements. Document prior receipt date in the authorization request.

Documentation Required

Required Baseline Documentation

Required baseline documentation must be obtained and submitted with the prior authorization request. Baseline values should be appropriate to the member's age and may include one or more of the following measurements.

Background

Hunter syndrome (Mucopolysaccharidosis II; MPS II) is an X‑linked lysosomal storage disorder caused by deficiency of iduronate‑2‑sulfatase (I2S). Elaprase (idursulfase) is an intravenous enzyme replacement therapy approved for treatment of the somatic (non‑CNS) manifestations of MPS II. Standard dosing is administered as regular infusions to address features such as reduced exercise tolerance, pulmonary impairment, organomegaly, joint stiffness, growth failure, and elevated urinary glycosaminoglycans; clinical studies have shown improvements in measures including the 6‑minute walk test and some pulmonary and organ size endpoints. Treatment carries risks including hypersensitivity and anaphylaxis and potential antibody formation, which are considered when evaluating continuation.

Definitions

Definitive diagnosis of MPS II (Hunter syndrome)

Enzyme assay criterionDeficient or absent iduronate-2-sulfatase (I2S) activity in white cells, fibroblasts, or plasma with normal activity of at least one other sulfatase

Molecular genetic criterionDetection of pathogenic mutation(s) in the IDS gene by molecular genetic testing

Acceptable specimen typesWhite cells, fibroblasts, or plasma for enzyme assay; DNA from blood or other validated specimen for IDS testing