CurrentMass General Brigham Health PlanPolicy N/A

Aldurazyme (laronidase) prior authorization for MPS I

Pharmacy benefit prior authorization policy for Aldurazyme (laronidase) covering commercial/exchange members, detailing eligibility, documentation and renewal criteria for treatment of mucopolysaccharidosis I (MPS I).

Policy Summary

PayerMass General Brigham Health Plan

PolicyAldurazyme (laronidase) prior authorization for MPS I

Policy CodePolicy N/A

Change TypeMaterial revisions — benefit moved to pharmacyspecialty designation added

Effective DateJan 1, 2026

Next Review DateN/A

Key ActionObtain prior authorization and ensure dispensing via a contracted specialty pharmacy; submit diagnostic confirmation of MPS I.

SourceLink

POLICY UPDATE CHANGES

Policy updated to reflect it no longer applies to the medical benefit and is pharmacy benefit only.

Program type set to Prior Authorization and medication designated specialty requiring dispensing via a contracted specialty pharmacy.

12 monthsInitial and reauthorization approval period

90 daysNew-to-plan continuity window

Commercial/ExchangePlan applicability

Pharmacy BenefitBenefit type

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Coverage Criteria for Aldurazyme (laronidase)

Initial Therapy

Covered when ALL of the following are met:

Initial authorization criteria: 1) Diagnosis of MPS I confirmed by enzyme assay demonstrating a deficiency of alpha-L-iduronidase activity and/or by genetic testing; 2) Member has the Hurler (severe MPS I) or Hurler-Scheie (attenuated MPS I) OR the member has the Scheie form (attenuated MPS I) with moderate to severe symptoms (examples: corneal clouding, joint stiffness, valvular heart disease).

Continuation Therapy

Reauthorization approved when ALL of the following are met:

Continuation criteria: Documentation of clinically positive response to therapy (e.g., improvement, stabilization, or slowing of disease progression).

Reauthorization granted for 12 months.

Use of Aldurazyme for central nervous system manifestations of mucopolysaccharidosis I (MPS I) has not been evaluated. Clinical benefit for CNS disease is unknown and therefore such manifestations are outside the scope of coverage determination.

The risks and benefits for mildly affected Scheie patients have not been established; Aldurazyme is indicated for patients with Hurler and Hurler-Scheie forms of MPS I and for Scheie patients only when they have moderate to severe symptoms

Initial Therapy — Detailed Criteria

Initial therapy

Initial authorization criteria

Initial: Confirmed diagnosis of MPS I by enzyme assay demonstrating deficiency of alpha-L-iduronidase activity and/or by genetic testing; Member has Hurler, Hurler-Scheie, or Scheie form with moderate to severe symptoms (e.g., corneal clouding, joint stiffness, valvular heart disease).

Continuation / Reauthorization Criteria

Reauthorization

Reauthorization requirements and duration

Reauthorization: Documentation of clinically positive response to therapy (improvement, stabilization, or slowing of disease progression).

Reauthorization granted for 12 months.

Provider Actions, Documentation & Authorization Rules

Prior Authorization
Prior Authorization Required
Prior authorization is required under the pharmacy benefit. This product is designated specialty and must be dispensed through a contracted specialty pharmacy.
Benefit: Pharmacy benefit
Program Type: Prior Authorization
Specialty dispensing required: fill at contracted specialty pharmacy
Pharmacy benefit contact: Phone 800-711-4555; Fax 844-403-1029

Note
New-to-Plan Continuity
Members new to the plan within the past 90 days may be granted authorization if they are currently receiving the requested medication. Authorization is not granted for therapy obtained only as samples or via manufacturer patient assistance programs.
New-to-plan window: 90 days
Exclusion: samples or manufacturer's patient assistance programs

Definitions and Diagnostic Requirements

Diagnostic confirmation — MPS I

Required diagnostic testDiagnosis of MPS I must be confirmed by enzyme assay demonstrating a deficiency of alpha-L-iduronidase enzyme activity and/or by genetic testing.

Applicable MPS I subtypesHurler, Hurler–Scheie, or Scheie with moderate to severe symptoms.

Examples of qualifying Scheie symptomsCorneal clouding, joint stiffness, valvular heart disease (indicative of moderate to severe symptoms).

CNS manifestation noteAldurazyme has not been evaluated for effects on central nervous system manifestations of MPS I.

Risk/benefit for mild ScheieRisks and benefits of treating mildly affected Scheie patients have not been established.

Background — Drug Indication and Context

Aldurazyme (laronidase) is an enzyme replacement therapy indicated for adult and pediatric patients with the Hurler and Hurler-Scheie forms of mucopolysaccharidosis I (MPS I), and for patients with the Scheie form who have moderate to severe symptoms.

The product has not been evaluated for effects on central nervous system manifestations of MPS I, and the risks and benefits for mildly affected Scheie patients are not established.

Site of Care and Dispensing Requirements

Billing Rule
Specialty pharmacy dispensing required
Aldurazyme is designated as a specialty medication and must be filled at a contracted specialty pharmacy per the pharmacy benefit.
Fill at contracted specialty pharmacy as required by specialty limitations
Benefit type: Pharmacy Benefit

Policy Summary

PayerMass General Brigham Health Plan

PolicyAldurazyme (laronidase) prior authorization for MPS I

Policy CodePolicy N/A

Change TypeMaterial revisions — benefit moved to pharmacyspecialty designation added

Effective DateJan 1, 2026

Next Review DateN/A

Key ActionObtain prior authorization and ensure dispensing via a contracted specialty pharmacy; submit diagnostic confirmation of MPS I.

SourceLink

On This Page

Step Therapy
Step Therapy
No step therapy requirement is listed for this medication under the plan.

Documentation Required
Required Documentation
Diagnostic confirmation is required to support approval. Submit documentation of enzyme assay and/or genetic testing demonstrating deficiency of alpha-L-iduronidase and clinical information supporting the MPS I subtype and symptom severity. For continuation, provide documentation of clinically positive response (improvement, stabilization, or slowing of disease progression).
Required diagnostic tests: enzyme assay demonstrating alpha-L-iduronidase deficiency and/or genetic testing
Required clinical documentation: MPS I subtype (Hurler, Hurler-Scheie, or Scheie with moderate–severe symptoms) and symptom details
Continuation requirement: documented clinically positive response to therapy

Denial Risk
Denial Triggers
Requests may be denied if diagnostic confirmation is lacking or if the member does not have a qualifying MPS I subtype or required symptom severity. Lack of documentation of prior therapy response for continuation requests may also trigger denial.
Denial trigger: No enzyme assay or genetic test confirming alpha-L-iduronidase deficiency
Denial trigger: Member does not meet qualifying MPS I subtype (Hurler, Hurler-Scheie, or Scheie with moderate–severe symptoms)
Denial trigger: For continuation, absence of documentation showing improvement, stabilization, or slowed disease progression