CurrentCignaPolicy N/A

Enzyme Replacement Therapy - Aldurazyme (laronidase) Coverage Criteria

This policy governs prior authorization, coverage criteria, dosing, and coding for Aldurazyme (laronidase) for treatment of Mucopolysaccharidosis type I (MPS I) for Cigna-administered health benefit plans and specifies who may prescribe and documentation required for approval.

Policy Summary

PayerCigna

PolicyEnzyme Replacement Therapy - Aldurazyme (laronidase) Coverage Criteria

Policy CodePolicy N/A

Change TypeDosing addedminor removals

Effective DateN/A

Next Review DateN/A

Key ActionObtain prior authorization and document diagnostic confirmation (enzyme assay or biallelic pathogenic/likely pathogenic IDUA variants) with prescription by or consultation with a relevant specialist.

SourceLink

POLICY UPDATE CHANGES

Removed: ONE of the following forms: Severe Mucopolysaccharidosis I (MPS I) or Attenuated.

Mucopolysaccharidosis I (MPS I) with moderate to severe symptoms — Added dosing.

No criteria changes across several update entries.

1 yearinitial approval duration

0.58 mg/kgmax dose per administration

weeklymax dosing frequency

J1931listed HCPCS code

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Prior Authauthorization requirement

Coverage Criteria for Aldurazyme (laronidase)

Mucopolysaccharidosis Type I (Hurler, Hurler-Scheie, Scheie)

Approve for 1 year if the patient meets BOTH of the following (A and B):

A AND B: A) Diagnosis established by ONE of the following (i or ii):

ONE of: i. Laboratory test demonstrating deficient α-L-iduronidase activity in leukocytes, fibroblasts, plasma, or serum.

ONE of: ii. Molecular genetic test demonstrating biallelic pathogenic or likely pathogenic IDUA gene (α-L-iduronidase) variants.

AND: B) Aldurazyme is prescribed by or in consultation with a geneticist, endocrinologist, a metabolic disorder sub-specialist, or a physician who specializes in the treatment of lysosomal storage disorders.

Approval duration = 1 year; dosing must not exceed 0.58 mg/kg IV no more frequently than once weekly.

Diagnostic confirmation of MPS I to support Aldurazyme therapy

Diagnostic confirmation (ONE of): Enzyme assay demonstrating deficient α-L-iduronidase activity in leukocytes, fibroblasts, plasma, or serum.

Diagnostic confirmation (ONE of): Molecular genetic testing demonstrating biallelic pathogenic or likely pathogenic variants in the IDUA gene.

Eligibility requirements: None at the top level. Coverage is determined by meeting the specific diagnostic and prescriber criteria for the FDA‑approved indication (MPS I) described in the Coverage Criteria.

Aldurazyme for any other use is considered not medically necessary. This policy does not cover uses of Aldurazyme outside the indications specified under the FDA‑approved MPS I criteria; criteria will be updated as new published data become available.

Use of Aldurazyme for indications other than MPS I (Hurler, Hurler‑Scheie, Scheie) is not medically necessary. Requests for Aldurazyme to treat conditions other than Mucopolysaccharidosis type I will be considered not covered under this policy.

Covered Indications

Diagnostic confirmation of MPS I to support Aldurazyme therapy

Diagnostic confirmation (ONE of): Enzyme assay demonstrating deficient α-L-iduronidase activity in leukocytes, fibroblasts, plasma, or serum.

Diagnostic confirmation (ONE of): Molecular genetic testing demonstrating biallelic pathogenic or likely pathogenic variants in the IDUA gene.

Coding and Billing

Considered Medically Necessary when criteria metHCPCSCovered

J1931

Injection, laronidase, 0.1 mg

Provider Actions and Authorization

Prior Authorization

Prior authorization required — approve for 1 year when criteria met

Prior authorization is required for benefit coverage of Aldurazyme; if the patient meets the policy diagnostic and prescriber criteria, approve for 1 year. Approvals may be extended if the patient continues to meet criteria and dosing requirements.

Prior authorization required for Aldurazyme (policy statement).
Approval duration = 1 year when BOTH diagnostic confirmation and prescriber criteria are met; extended approvals allowed if criteria continue to be met.

Step Therapy

Step therapy context — HSCT discussed but not mandated

HSCT is identified as a treatment option for severe forms of MPS I in select patients (children <2 years who are cognitively intact), but the policy does not impose a formal step therapy sequence requiring HSCT before Aldurazyme.

HSCT is indicated for severe forms of MPS I in children <2 years who are cognitively intact.

Not Covered / Exclusions

Genetic or enzyme testing performed solely to support use of Aldurazyme for non‑MPS I indications does not render the treatment medically necessary and would not make therapy covered. Aldurazyme is considered not medically necessary for any use outside of MPS I (Hurler, Hurler‑Scheie, Scheie), and diagnostic testing for non‑MPS I indications will not change that coverage stance.

Background and Disease Overview

Mucopolysaccharidosis type I (MPS I) is a rare autosomal recessive lysosomal storage disorder caused by deficiency of α‑L‑iduronidase, resulting in accumulation of glycosaminoglycans and progressive multi‑organ dysfunction. Clinical manifestations can include characteristic facial features, corneal clouding, cardiomyopathy, hepatosplenomegaly, respiratory compromise, dysostosis multiplex, joint stiffness, and variable cognitive impairment. Definitive diagnosis is established by demonstrating deficient α‑L‑iduronidase activity in leukocytes, fibroblasts, plasma, or serum, or by identifying biallelic pathogenic or likely pathogenic variants in the IDUA gene.

Definitive diagnosis of MPS I — acceptable diagnostic methods

Enzyme activity assayDemonstration of deficient α-L-iduronidase activity in leukocytes, fibroblasts, plasma, or serum.

Molecular genetic testingIdentification of biallelic pathogenic or likely pathogenic variants in the IDUA gene (molecular genetic test).

Sample types for enzyme assayEnzyme activity testing may be performed on leukocytes, fibroblasts, plasma, or serum.

Clinical classification noteMPS I includes Hurler, Hurler-Scheie, and Scheie forms — all result from the same α-L-iduronidase deficiency; definitive diagnosis requires enzyme assay or biallelic IDUA variants.

Policy Summary

PayerCigna

PolicyEnzyme Replacement Therapy - Aldurazyme (laronidase) Coverage Criteria

Policy CodePolicy N/A

Change TypeDosing addedminor removals

Effective DateN/A

Next Review DateN/A

SourceLink

On This Page

No formal step therapy sequencing is imposed by this policy.

Documentation Required

Required documentation — diagnostic confirmation and specialist involvement

Document diagnostic confirmation of MPS I by either an enzyme assay showing deficient α-L-iduronidase activity (leukocytes, fibroblasts, plasma, or serum) or molecular testing demonstrating biallelic pathogenic/likely pathogenic IDUA variants, and document that Aldurazyme is prescribed by or in consultation with an appropriate specialist.

Acceptable diagnostic confirmation: laboratory demonstration of deficient α-L-iduronidase activity in leukocytes, fibroblasts, plasma, or serum; OR molecular genetic test showing biallelic pathogenic/likely pathogenic IDUA variants.
Prescriber documentation: prescribed by or in consultation with a geneticist, endocrinologist, metabolic disorder sub‑specialist, or physician specializing in lysosomal storage disorders.

Note

Prescriber requirement — specialist involvement required

Aldurazyme must be prescribed by or in consultation with a specialist such as a geneticist, endocrinologist, metabolic disorder sub‑specialist, or a physician who specializes in lysosomal storage disorders.

Specialist prescriber or documented consultation required for approval.

Denial Risk

Denial triggers — missing codes, prior auth, or documentation

Claims submitted for services that are not accompanied by covered diagnosis or procedure codes listed in the policy will be denied as not covered; requests without prior authorization or lacking required documentation are subject to denial.

Claims billed with codes not covered by this policy are not eligible for reimbursement.
Requests submitted without prior authorization will be denied.
Requests missing required diagnostic or prescriber documentation may be denied.

Documentation requirementDiagnostic confirmation (enzyme assay or molecular testing showing biallelic pathogenic/likely pathogenic IDUA variants) must be documented to support coverage.