CurrentAvMedPolicy Pub. 100-2

Prior authorization/step-edit for Nexviazyme, Pombiliti + Opfolda

Form and clinical criteria used by AvMed to authorize medical benefit administration of Nexviazyme (avalglucosidase alfa), Pombiliti (cipaglucosidase alfa) with Opfolda (miglustat), including initial and reauthorization requirements for members with Pompe disease.

Policy Summary

PayerAvMed

PolicyPrior authorization/step-edit for Nexviazyme, Pombiliti + Opfolda

Policy CodePolicy Pub. 100-2

Change TypeNo material changes

Effective Date

Next Review Date

Key ActionPrescriber must complete and sign the AvMed medical prior authorization/step-edit request form to obtain authorization.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

12 monthsInitial authorization duration

12 monthsReauthorization duration

2300 mg/14dMax IV units

8 caps/28dOpfolda dispensing

Coverage Criteria

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Age and product-specific age/weight: Member meets ONE of the following age requirements: Nexviazyme: >=1 year; Pombiliti: >=18 years and >=40 kgage/weight

Diagnosis: Member has a diagnosis of late-onset (non-infantile) Pompe disease (acid alpha-glucosidase (GAA) deficiency) confirmed by ONE of: deficiency of GAA enzyme activity <40% of lab-specific normal mean OR detection of biallelic pathogenic GAA variantsdiagnostic criteria

Baseline functional measures: Member has documented baseline percent predicted forced vital capacity (FVC) and/or 6-minute walk test (6MWT); supporting labs/measurements must be submittedbaseline FVC and/or 6MWT documented

Please submit labs

Prior ERT failure or lack of improvement: Member has NOT demonstrated an improvement in objective measures (e.g., FVC, 6MWT) after receiving ONE of the following for at least one year: Lumizyme (alglucosidase alfa) IV infusion OR Nexviazyme (avalglucosidase alfa-ngpt) IV infusion>=1 year prior ERT without improvement

Please submit chart notes documenting prior ERT duration and response; applies prior to Pombiliti approval per checklist

Treatment combination and exclusivity: Requested medication will NOT be used in combination with other enzyme replacement therapies (e.g., Lumizyme); for Pombiliti requests, medication will be used in combination with Opfolda (miglustat) and provider must select requested Opfolda access pathway (Pharmacy benefit or Medical Buy & Bill)combination/exclusivity

If Opfolda access selected, choose Pharmacy Benefit or Medical Buy & Bill

inv-02: Reauthorization

Covered when ALL of the following are met

Continuation of initial criteria: Member continues to meet all initial authorization criteriacontinue initial criteria

Tolerability: Member has experienced an absence of unacceptable toxicity from the drug (e.g., anaphylaxis, severe hypersensitivity reactions, severe infusion-associated reactions, acute cardiorespiratory failure)no unacceptable toxicity

Clinical benefit: Member has demonstrated a beneficial response to therapy compared to pretreatment baseline in one or more of: disease stabilization, improvement in FVC, improvement in 6MWT; supporting labs/measurements must be submittedobjective improvement or stabilization

Please submit labs/measurements

Antibody monitoring: Member is being monitored for antibody formation (including neutralizing antibodies)antibody monitoring in place

Coding

Medical benefit drug HCPCSHCPCSCovered

J0219

Nexviazyme (avalglucosidase alfa-ngpt)

Medical benefit drug HCPCS (Pombiliti + Opfolda)HCPCSCovered

J1203	Pombiliti (cipaglucosidase alfa-atga)
J1202	Opfolda (miglustat) - medical benefit pathway

inv-07: Maximum IV product units

Maximum IV product units2300 mg every 14 days

Applies toNexviazyme and Pombiliti (IV products)

Dosing intervalEvery 14 days (biweekly)

inv-08: Opfolda dispensing when Pombiliti approved

Opfolda dispensing when Pombiliti approvedUp to 8 capsules per 28 days

Access pathwaysPharmacy Benefit or Medical Buy & Bill (select on form)

NoteSubsequent authorization entered to allow Opfolda at this quantity when Pombiliti approval granted

Provider Actions & Requirements

Prior Authorization

Completed signed PA form required

The prescribing physician must complete and sign the AvMed prior authorization/step-edit request form. Preprinted signature stamps are not acceptable. If the form is incomplete, illegible, or unsigned, authorization may be delayed or denied. Ensure phone and fax numbers are correct on the form; missing or incorrect contact information can delay processing.

Unsigned or incomplete forms risk denial or processing delays.
Provide accurate phone and fax contact information on the request form.
Fax completed form to 1-844-668-1550.

Step Therapy

Step-edit / prior ERT requirement for Pombiliti

Pombiliti (cipaglucosidase alfa-atga) requires prior step-edit evidence of failure or lack of improvement on prior enzyme replacement therapy (ERT) before approval. The member must be ≥18 years old, weigh at least 40 kg, have late-onset Pompe disease, and meet diagnostic confirmation criteria. Documentation must show at least one year of prior ERT (e.g., Lumizyme or Nexviazyme) without improvement in objective measures.

Prior ERT tried for ≥1 year with documentation of lack of improvement (e.g., FVC, 6MWT).
Diagnostic confirmation via reduced GAA enzyme activity <40% of lab normal mean or biallelic pathogenic GAA variants.
Pombiliti must be prescribed with Opfolda; indicate pharmacy or medical buy & bill pathway.

Documentation Required

Required supporting documentation

All requests must include supporting documentation: baseline and follow-up objective measures, relevant labs, and chart notes. Failure to submit required documentation may result in denial.

Baseline percent predicted FVC and/or 6MWT results (submit lab/diagnostic reports).
Chart notes documenting duration and response to prior ERT (including dates showing ≥1 year of therapy).
Documentation of diagnosis method: GAA enzyme activity result (<40% of lab normal mean) or molecular genetic testing showing biallelic pathogenic variants.
For reauthorization: objective evidence of benefit (stabilization or improvement in FVC or 6MWT) and monitoring for antibody formation.

Denial Risk

Incomplete information or missing documentation

Incomplete, missing, or illegible information (including unsigned forms, absent diagnostic test results, missing chart notes, or lack of prior therapy documentation) may delay processing or result in denial. Provide complete clinical details, dates, weights, dosing schedule, and contact information to avoid delays.

Missing baseline FVC or 6MWT data risks denial.
Absent documentation of ≥1 year prior ERT for Pombiliti requests risks denial.
Unsigned prescriber signature will invalidate the request.

Background

These products include intravenous enzyme replacement therapies—Nexviazyme (avalglucosidase alfa) and Pombiliti (cipaglucosidase alfa)—and the oral disease modifier Opfolda (miglustat). They are indicated for the treatment of late-onset (non‑infantile) Pompe disease (acid alpha‑glucosidase deficiency); authorization requires diagnostic confirmation and baseline functional measures such as percent predicted FVC and/or the 6‑minute walk test, and when Pombiliti is approved it must be used in combination with Opfolda per the checklist.

Definitions

inv-14: Confirmed Pompe disease

DefinitionConfirmed Pompe disease is established by ONE of: deficient GAA enzyme activity <40% of the lab-specific normal mean OR detection of biallelic pathogenic GAA variants

Required documentationSubmit lab results showing percent of lab normal mean or molecular genetic testing report

Clinical contextDiagnosis pertains to late-onset (non-infantile) Pompe disease (acid alpha-glucosidase deficiency)

Initial Therapy Criteria

inv-15: Initial Therapy

Initial authorization criteria

Age/weight: Nexviazyme: >=1 year; Pombiliti: >=18 years and >=40 kgage/weight

Diagnostic confirmation: GAA enzyme activity <40% of lab-specific normal mean OR detection of biallelic pathogenic GAA variantsdiagnostic tests

Baseline measures: Documented baseline percent predicted FVC and/or 6MWT (please submit labs)baseline FVC/6MWT

Please submit labs/measurements

Continuation / Reauthorization Criteria

inv-16: Continuation Therapy

Reauthorization requirements

See Reauthorization nodes: Member continues initial criteria, has absence of unacceptable toxicity, demonstrates benefit or stabilization, and is monitored for antibodiessee reauthorization

Submit documentation (labs, diagnostics, chart notes) to support each checked item

Step Therapy

Step	Requirement	Notes
1	Must document prior enzyme replacement therapy (ERT) of at least one year with either Lumizyme (alglucosidase alfa) IV infusion or Nexviazyme (avalglucosidase alfa-ngpt) IV infusion	Chart notes must be submitted documenting >=1 year of prior ERT and that the member has NOT demonstrated improvement in objective measures (FVC and/or 6MWT)
2	Requested Pombiliti (cipaglucosidase alfa) will be used in combination with Opfolda (miglustat) and will NOT be used with other ERTs	Provider must select Opfolda access pathway (Pharmacy benefit or Medical Buy & Bill) when requesting Pombiliti

Site of Care

Note

Specify infusion center/office or choose specialty pharmacy; provide NPI/DEA

Indicate the location/site of drug administration on the form and provide the NPI or DEA number of the administering location, or select the specialty pharmacy option (Proprium Rx).

Provide administering location and NPI or DEA #, OR
Select Specialty Pharmacy - Proprium Rx

Policy Summary

PayerAvMed

PolicyPrior authorization/step-edit for Nexviazyme, Pombiliti + Opfolda

Policy CodePolicy Pub. 100-2

Change TypeNo material changes

Effective Date

Next Review Date

Key ActionPrescriber must complete and sign the AvMed medical prior authorization/step-edit request form to obtain authorization.

SourceLink

On This Page

OpenPayer

Prior authorization/step-edit for Nexviazyme, Pombiliti + Opfolda

Coverage Criteria

Coding

Provider Actions & Requirements

Background

Definitions

Initial Therapy Criteria

Continuation / Reauthorization Criteria

Step Therapy

Quantity Limits

Site of Care