CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Xenpozyme (olipudase alfa) — coverage criteria for non‑CNS ASMD

Covers clinical criteria, dosing, authorization, and renewal requirements for Xenpozyme (olipudase alfa) for treatment of non‑CNS manifestations of acid sphingomyelinase deficiency (ASMD) for Neighborhood Health Plan of Rhode Island members (Medicaid, Commercial, Medicare).

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyXenpozyme (olipudase alfa) — coverage criteria for non‑CNS ASMD

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateAug 1, 2023

Next Review DateN/A

Key ActionObtain confirmatory biallelic SMPD1 testing or enzyme activity assay (<10% of controls) and document baseline DLco/PFTs, spleen and liver volumes, platelet count, and current weight prior to authorization.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

6 monthsInitial auth length

340 mg / 14 daysMax dose per interval

J0218HCPCS code

E75.241, E75.244Covered ICD-10

Week 14–16Maintenance reached

Related Billing Codes

HCPCS-J0218-OLIPUDASE-ALFA-INJECTION-1MG

HCPCS J0218: Injection, olipudase alfa, 1 mg

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Coverage Criteria for Xenpozyme (olipudase alfa)

inv-01: Initial Approval Criteria

Covered when ALL of the following are met

Clinical indication and diagnosis: Patient has a definitive diagnosis of acid sphingomyelinase deficiency (ASMD) confirmed by detection of biallelic pathogenic SMPD1 mutations by molecular genetic testing OR acid sphingomyelinase enzyme activity <10% of controls measured in peripheral leukocytes, cultured fibroblasts, or lymphocytes; AND therapy is intended for non‑CNS manifestations of ASMD.

Xenpozyme is not expected to cross the blood‑brain barrier or treat CNS manifestations.

Baseline and monitoring: Documented baseline measures prior to treatment initiation: percent predicted DLco or other age‑appropriate pulmonary function testing, spleen volume, liver volume, plasma lyso‑sphingomyelin, and/or platelet count; for pediatric patients, height Z‑score and skeletal maturation; baseline ALT and AST within 1 month prior to initiation and within 72 hours prior to any infusion during dose escalation; and documentation of current weight (kg).

These baseline measures are necessary for renewal assessment.

Prescriber and dosing: Medication is prescribed by or in consultation with a specialist familiar with lysosomal storage disorders; dose does not exceed 3 mg/kg every 2 weeks; documentation of patient weight (kg) for dosing calculations.3 mg/kg every 2 weeks maximum

Follow weight‑based escalation schedule to maintenance per product labeling.

Reproductive precautions: Females of reproductive potential must have pregnancy status verified prior to start of therapy and must use effective contraception during treatment and for 14 days after the last dose if therapy is discontinued.

Exclusion criteria: Coverage will not be provided if any of the following are present: acute or rapidly progressive neurologic abnormalities; requirement for invasive ventilatory support or non‑invasive ventilatory support while awake >12 hours/day; platelet count <60 × 10^3/µL; INR >1.5; ALT or AST >250 IU/L or total bilirubin >1.5 mg/dL.

Medicare members previously treated within past 365 days are not subject to step therapy requirements.

inv-02: Renewal Criteria

Renewal allowed when ALL of the following are met

Continuing eligibility: Patient continues to meet all initial approval criteria and other relevant indication‑specific requirements.

Renewal period every 6 months.

Safety and toxicity: Absence of unacceptable toxicity related to therapy (examples include anaphylaxis, severe hypersensitivity or infusion‑associated reactions, severely elevated liver transaminases).

Neurologic status: Patient has not experienced progressive or irreversible severe cognitive impairment since initiation of therapy.

Demonstration of disease response (one or more): Improvement or stability from pre‑treatment baseline in at least one of the following: percent predicted DLco or other age‑appropriate PFTs; spleen and/or liver volumes; reduction in plasma lyso‑sphingomyelin; platelet count; or improvement in linear growth (mean height Z‑scores) for pediatric patients.

Coverage will not be provided for requests involving acute or rapidly progressive neurologic abnormalities. Members who require invasive ventilatory support or who require non‑invasive ventilatory support while awake for >12 hours/day are excluded. Additional clinical exclusion thresholds include platelet count <60 × 10^3/µL, INR >1.5, or markedly abnormal liver tests—ALT or AST >250 IU/L or total bilirubin >1.5 mg/dL. These conditions must be evaluated and documented prior to initiation, as they preclude coverage.

Xenpozyme (olipudase alfa) is indicated and covered only for non‑CNS manifestations of ASMD. Use of therapy for primary CNS manifestations (e.g., Niemann‑Pick disease type A / infantile neurovisceral disease) is not supported because the product is not expected to cross the blood‑brain barrier. Requests or testing solely directed at CNS disease manifestations are therefore not covered under this policy and will be evaluated as such.

Diagnosis and Indications

inv-17: Diagnostic confirmation of ASMD to determine eligibility for Xenpozyme therapy

Diagnostic confirmation required to determine eligibility for Xenpozyme therapy

Diagnostic methods (one required): Detection of biallelic pathogenic mutations in the SMPD1 gene by molecular genetic testing; OR deficiency of acid sphingomyelinase enzyme activity <10% of controls measured in peripheral leukocytes, cultured fibroblasts, or lymphocytes.enzyme activity <10% of controls

Confirmatory molecular testing or enzyme assay is required prior to coverage; therapy is for non‑CNS manifestations only.

Billing and Coding

Covered HCPCS/CPT CodesHCPCSCovered

J0218

Injection, olipudase alfa-rpcp, 1 mg; 1 billable unit = 1 mg

NDCNDC

58468-0050-xx

Xenpozyme 20 mg lyophilized powder for reconstitution in a single-dose vial

Covered Diagnosis CodesICD-10Covered

E75.241	Niemann-Pick disease type B
E75.244	Niemann-Pick disease type A/B

Maximum HCPCS units per dosing interval

Maximum HCPCS units per dosing interval340 billable units (340 mg) every 14 days

HCPCS unit definition1 billable unit = 1 mg (see J0218 for billing)

Dosing intervalDoses billed on a 14-day (every 2 weeks) schedule

Provider Requirements, Authorization, and Documentation

Prior Authorization

Authorization Period

Coverage will be provided for an initial authorization period of 6 months. Authorization may be renewed in 6-month increments when renewal criteria are met.

Initial authorization duration: 6 months
Renewal interval: may be renewed every 6 months

Step Therapy

Step Therapy Exception

Medicare members who have received this medication within the past 365 days are exempt from step therapy requirements.

Step therapy exception applies only to Medicare members with prior receipt within 365 days

Documentation Required

Member Eligibility

High‑level eligibility for Xenpozyme requires a definitive diagnosis of ASMD confirmed by either biallelic pathogenic SMPD1 mutations on molecular testing or acid sphingomyelinase enzyme activity <10% of controls measured in peripheral leukocytes, cultured fibroblasts, or lymphocytes. Therapy is for the treatment of non‑CNS manifestations of ASMD and must be prescribed by, or in consultation with, a specialist experienced in lysosomal storage disorders.

Definitions and Diagnostic Criteria

ASMD diagnosis

Definition of ASMD (diagnosis)Definitive diagnosis of acid sphingomyelinase deficiency (ASMD; Niemann‑Pick disease) confirmed per document criteria

Required diagnostic evidenceDetection of biallelic pathogenic SMPD1 mutations OR acid sphingomyelinase enzyme activity <10% of controls

Clinical phenotype requirementPatient has a clinical diagnosis consistent with NPD‑B or NPD‑A/B and therapy is for non‑CNS manifestations

Diagnostic criteria

Molecular genetic testing criterionDetection of biallelic pathogenic mutations in the SMPD1 gene confirms diagnosis

Enzyme assay diagnostic thresholdAcid sphingomyelinase enzyme activity <10% of controls measured in peripheral leukocytes, cultured fibroblasts, or lymphocytes

Non‑Covered Indications

Testing or treatment directed solely at CNS manifestations of ASMD (for example, management of infantile neurovisceral disease / NPD‑A) is not a covered indication for Xenpozyme because the therapy is not expected to treat central nervous system disease.

Background

Xenpozyme (olipudase alfa) is an enzyme replacement therapy indicated for the treatment of non‑CNS manifestations of acid sphingomyelinase deficiency (ASMD). Clinical trial evidence demonstrated benefit for systemic disease measures (for example, improvements in DLco, spleen and liver volumes, platelet count, and pediatric height Z‑scores). The agent is not expected to cross the blood‑brain barrier, and therefore does not treat CNS manifestations of ASMD.