CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Xenpozyme (olipudase alfa) Intravenous — Coverage Criteria for ASMD

Coverage policy for intravenous olipudase alfa (Xenpozyme) for non-CNS manifestations of acid sphingomyelinase deficiency (ASMD) for Medicaid, Commercial, and Medicare members of Neighborhood Health Plan of Rhode Island.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyXenpozyme (olipudase alfa) Intravenous — Coverage Criteria for ASMD

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateAug 1, 2023

Next Review DateN/A

Key ActionObtain prior authorization for initial approval (6 months), with required baseline measures documented before initiation.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

6 moinitial authorization duration

340 mgmax billable units per dose

J0218HCPCS billing code

E75.241, E75.244Covered ICD-10 diagnoses

Not for CNSNot for CNS disease

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Coverage Criteria for Xenpozyme (olipudase alfa)

inv-01: Initial Approval

Covered when ALL of the following are met:

Initial approval criteria: 1) Definitive diagnosis of ASMD by either 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; 2) Clinical diagnosis consistent with Niemann-Pick disease type B (NPD-B) or type A/B (NPD-A/B) for non‑CNS manifestations (note: infantile neurovisceral ASMD/NPD-A was not studied); 3) Documented baseline measures necessary for renewal: percent predicted DLco or other age‑appropriate pulmonary function testing, spleen volume, liver volume, plasma lyso‑sphingomyelin, and/or platelet count; pediatric patients only: height Z‑score and skeletal maturation; 4) Baseline transaminases (ALT and AST) within 1 month prior to treatment initiation, within 72 hours prior to any infusion dose escalation, and monitored periodically during therapy; 5) Medication prescribed by, or in consultation with, a specialist familiar with treatment of lysosomal storage disorders; 6) Dose does not exceed 3 mg/kg every 2 weeks; 7) Documentation of current patient weight (kg); 8) Females of reproductive potential have pregnancy status verified and will use effective contraception during treatment and for 14 days after the last dose.

inv-02: Renewal Criteria

Renewal approval requires ALL of the following:

Renewal criteria: 1) Continued meeting of initial and other indication‑specific relevant criteria (e.g., concomitant requirements, performance status); 2) Absence of unacceptable toxicity (examples include anaphylaxis, severe hypersensitivity, severe infusion‑associated reactions, or severely elevated liver transaminases); 3) No progressive or irreversible severe cognitive impairment; 4) Demonstrated disease response or stability compared with pre‑treatment baseline as shown by one or more of: improvement or stability in percent predicted DLco or other age‑appropriate PFTs; improvement or stability in spleen and/or liver volumes; reduction in plasma lyso‑sphingomyelin; improvement or stability in platelet count; or improvement in linear growth (mean height Z‑scores) for pediatric patients.

Requests for treatment of acute or rapidly progressive neurologic abnormalities (CNS manifestations) are excluded. The policy states that Xenpozyme is not expected to cross the blood–brain barrier and will be used for non‑CNS manifestations only, therefore treatments intended to address CNS disease are not covered.

Therapy is not medically necessary if the prescribed dose exceeds 3 mg/kg every 2 weeks. Coverage will also be denied when other exclusion conditions listed in the policy apply (see conditions triggering non‑coverage such as acute neurologic decline, need for prolonged ventilatory support, markedly abnormal INR or transaminases, or pregnancy/contraception requirements).

Covered Indications and Diagnostic Confirmation

inv-15: Confirmation of ASMD to establish eligibility for Xenpozyme therapy

Definitive diagnosis of ASMD may be established by either of the following:

ONE of: Detection of biallelic pathogenic mutations in the SMPD1 gene by molecular genetic testing.

ONE of: Deficiency of acid sphingomyelinase enzyme activity <10% of controls as measured in peripheral leukocytes, cultured fibroblasts, or lymphocytes.

Eligibility Requirements

Eligible patients must have a definitive diagnosis of ASMD confirmed by either detection of biallelic pathogenic SMPD1 variants by molecular genetic testing or by acid sphingomyelinase enzyme activity <10% of controls. The patient must have a clinical diagnosis consistent with Niemann‑Pick disease type B or A/B and documented baseline measures (e.g., percent predicted DLco or appropriate pulmonary function testing, spleen and liver volumes, plasma lyso‑sphingomyelin, platelet count).

Additional eligibility requirements include: prescription by or in consultation with a specialist familiar with lysosomal storage disorders; documentation of current weight (kg); baseline ALT and AST within 1 month before initiation and within 72 hours prior to any infusion dose escalation; and, for females of reproductive potential, verified pregnancy status and use of effective contraception during treatment and for 14 days after the last dose.

Provider Actions, Documentation, and Prior Authorization

Prior Authorization

Prior Authorization Required

Prior authorization is required. Initial approval is granted for 6 months; renewals may be authorized in 6-month increments. For Medicare members, Neighborhood will follow any applicable NCD/LCD; if an NCD/LCD exists that governs coverage, the Medicare determination takes precedence. Medicare members who have received this medication within the past 365 days are exempt from Step Therapy requirements.

Initial authorization duration: 6 months
Renewal: every 6 months
Medicare NCD/LCD override: applicable NCD/LCDs take precedence

Step Therapy

Step Therapy Considerations

Step therapy does not apply to Medicare members who have documentation of prior treatment with this medication within the past 365 days. For non‑Medicare members, any step therapy requirements in the policy should be followed unless overridden by Medicare NCD/LCD guidance.

Medicare exemption: prior treatment within 365 days exempts patient from step therapy
Non‑Medicare members: follow step therapy rules in policy when applicable

Denial Risk

Conditions Triggering Non‑Coverage

Coverage will be denied when any of the following are present: acute or rapidly progressive neurologic abnormalities; invasive ventilatory dependence OR requirement for non‑invasive ventilatory support while awake for >12 hours/day; International Normalized Ratio (INR) > 1.5; alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 250 IU/L; total bilirubin ≥ 71.5 mg/dL. Ensure these exclusion conditions are documented; requests meeting any of these conditions should be denied.

Acute or rapidly progressive neurologic abnormalities — not covered
Invasive ventilatory support OR non‑invasive ventilatory support while awake >12 hours/day — not covered
INR > 1.5 — not covered
ALT or AST > 250 IU/L — not covered
Total bilirubin ≥ 71.5 mg/dL — not covered

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 ICD-10 CodesICD-10Covered

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

inv-08: Maximum dose per billing — 340 billable units (340 mg) every 14 days

Maximum billable units340 billable units (340 mg) per dose

Billing frequencyEvery 14 days

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

Not Covered Uses

Use of Xenpozyme for CNS manifestations of ASMD or for infantile neurovisceral ASMD (NPD‑A) was not studied and is not expected to be covered.

Background

Acid sphingomyelinase deficiency (ASMD; Niemann‑Pick disease types B and A/B) is a lysosomal storage disorder caused by deficient acid sphingomyelinase activity leading to non‑CNS manifestations such as hepatosplenomegaly, pulmonary dysfunction, thrombocytopenia, and growth impairment in children. Olipudase alfa (Xenpozyme) is an enzyme replacement therapy indicated for non‑CNS manifestations of ASMD and has demonstrated improvement or stabilization in DLco, spleen and liver volumes, platelet counts, and pediatric height Z‑scores in clinical trials.

Definitions

inv-14: Definitive diagnosis of ASMD — biallelic SMPD1 pathogenic variants OR enzyme activity <10% of control

Definitive diagnostic optionsDetection of biallelic pathogenic mutations in the SMPD1 gene by molecular genetic testing

Alternative diagnostic optionAcid sphingomyelinase enzyme activity <10% of controls measured in peripheral leukocytes, cultured fibroblasts, or lymphocytes

Clinical correlation requirementClinical diagnosis consistent with Niemann-Pick disease type B (NPD‑B) or A/B (NPD‑A/B) is required