CurrentMass General Brigham Health PlanPolicy N/A

Xenpozyme (olipudase alfa-rpcp) — Coverage Criteria for Non‑CNS ASMD

Policy governing prior authorization, quantity/step limits, and medical benefit handling for Xenpozyme (olipudase alfa) for treatment of non‑CNS manifestations of acid sphingomyelinase deficiency (ASMD) for Mass General Brigham Health Plan members.

Policy Summary

PayerMass General Brigham Health Plan

PolicyXenpozyme (olipudase alfa-rpcp) — Coverage Criteria for Non‑CNS ASMD

Policy CodePolicy N/A

Change TypeNo material change

Effective DateMar 1, 2023

Next Review DateN/A

Key ActionObtain prior authorization and document diagnosis confirmation (enzyme deficiency or SMPD1 mutation) and clinical response for reauthorization.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

2023-03-01policy effective date

Approvedindication

12 moapproval duration

Coverage Criteria for Xenpozyme (olipudase alfa)

Initial Authorization

Coverage may be granted either for members new to the plan already receiving Xenpozyme, or when ALL of the following are met:

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Existing therapy option: Member is new to the plan and currently receiving treatment with Xenpozyme (excluding product obtained as samples or via manufacturer patient assistance programs)

Alternative pathway to meet authorization

Diagnosis confirmation: Member has a diagnosis of non‑CNS manifestations of acid sphingomyelinase deficiency (ASMD) AND ONE of: documented deficiency of acid sphingomyelinase (measured in peripheral leukocytes, cultured fibroblasts, or lymphocytes) OR genetic testing documenting an SMPD1 mutation

Both clinical diagnosis and laboratory/genetic confirmation required

Initial Therapy

Initial authorization pathways:

New-to-plan current treatment: Member is new to the plan and currently receiving treatment with Xenpozyme (excluding product obtained as samples or via manufacturer patient assistance programs)

Alternate pathway to full initial criteria

Diagnostic confirmation: Member has non‑CNS ASMD confirmed by ONE of the following: documented deficiency of acid sphingomyelinase (measured in peripheral leukocytes, cultured fibroblasts, or lymphocytes) OR genetic testing documenting a mutation in the SMPD1 gene

Required for new starts

Requests from members who are new to the plan and currently receiving treatment with Xenpozyme may qualify for the new-to-plan current treatment authorization pathway. However, use that was obtained only as samples or provided through manufacturer patient assistance programs is explicitly excluded from this pathway and cannot be relied on to meet the ‘‘currently receiving treatment’’ criterion.

Initial Therapy

Initial authorization pathways:

New-to-plan current treatment: Member is new to the plan and currently receiving treatment with Xenpozyme (excluding product obtained as samples or via manufacturer patient assistance programs)

Alternate pathway to full initial criteria

Required for new starts

Continuation / Reauthorization

Continuation of Therapy

Reauthorization is granted when ALL of the following are documented:

Clinical response: Provider documents member is responding to therapy (e.g., improvement in lung function; reduction in spleen volume; reduction in liver volume; improvement in platelet count; improvement in linear growth progression)

Objective or clinical improvements required for continuation

Reauthorization

Continuation (reauthorization) requirements and duration:

Response documented: Provider documents clinical response to therapy as evidence for reauthorization (improvement in lung function, reduction in spleen and/or liver volume, improvement in platelet count, or improvement in linear growth)

Reauthorizations granted for 12 months

Provider Actions and Documentation Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is required for Xenpozyme under the pharmacy benefit and specialty medication pathways. Contact the phone/fax numbers below to request authorization.

All Plans Pharmacy/Specialty: Phone: 877-519-1908; Fax: 855-540-3693
Non-Specialty Medications All Plans: Phone: 800-711-4555

Step Therapy

Program Type / Benefit Notes

Step therapy and quantity limit program types are listed for this product. There are no additional step requirements specified in this section; follow any step therapy or quantity limit rules in the plan benefit.

Program Type: Quantity Limit
Benefit: Step Therapy
Medical Benefit: Specialty Limitations: N/A

Documentation Required

Diagnosis Confirmation Required

Documentation must include confirmation of the diagnosis. Acceptable evidence is a documented deficiency of acid sphingomyelinase or genetic testing showing an SMPD1 mutation. Include supporting lab and genetic reports with the authorization request.

Diagnosis required: non-CNS manifestations of acid sphingomyelinase deficiency (ASMD)
Acceptable confirmation: documented acid sphingomyelinase deficiency measured in peripheral leukocytes, cultured fibroblasts, or lymphocytes
Or: genetic testing documenting a mutation in SMPD1

Denial Risk

New-to-Plan / Denial Risk

Requests may be denied when the member is not new to the plan and is not currently receiving treatment with Xenpozyme. Coverage exceptions do not apply when the product was obtained only as samples or through manufacturer's patient assistance programs.

Authorization may be granted for members new to plan who are currently receiving Xenpozyme — excludes samples or manufacturer patient assistance program supplies

Program type	Defined step requirement
Step Therapy	Not specified in policy body — no specific step requirements are defined for Xenpozyme in the coverage criteria; step therapy is listed only as a program type in the header.

Definitions

ASMD / non‑CNS manifestations — definition and diagnostic criteria

TermAcid sphingomyelinase deficiency (ASMD)

DefinitionA genetic disorder caused by mutations in the SMPD1 gene resulting in deficient acid sphingomyelinase activity; therapy targets non‑central nervous system (non‑CNS) manifestations.

Diagnostic confirmation acceptedDocumented deficiency of acid sphingomyelinase measured in peripheral leukocytes, cultured fibroblasts, or lymphocytes OR genetic testing documenting an SMPD1 mutation.

Indication populationApproved for treatment of non‑CNS manifestations of ASMD in adult and pediatric patients.

Background

Xenpozyme (olipudase alfa) is an enzyme replacement therapy indicated for the treatment of non‑central nervous system (non‑CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients. Clinical response used to support continuation includes improvements such as enhanced lung function, reductions in spleen and liver volumes, improved platelet counts, or gains in linear growth progression.

Policy Summary

PayerMass General Brigham Health Plan

PolicyXenpozyme (olipudase alfa-rpcp) — Coverage Criteria for Non‑CNS ASMD

Policy CodePolicy N/A

Change TypeNo material change

Effective DateMar 1, 2023

Next Review DateN/A

Key ActionObtain prior authorization and document diagnosis confirmation (enzyme deficiency or SMPD1 mutation) and clinical response for reauthorization.

SourceLink

On This Page

OpenPayer

Xenpozyme (olipudase alfa-rpcp) — Coverage Criteria for Non‑CNS ASMD

Coverage Criteria for Xenpozyme (olipudase alfa)

Initial Therapy

Continuation / Reauthorization

Provider Actions and Documentation Requirements

Site of Care / Benefit

Quantity Limits and Drug Listing

Step Therapy

Definitions

Background