Avalglucosidase Alfa-ngpt (Nexviazyme) Form

Avalglucosidase alfa-ngpt (Nexviazyme™) is a hydrolytic lysosomal glycogen-specific enzyme. FDA Approved Indication(s) Nexviazyme is indicated for the treatment of patients 1 year of age and older with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency). Policy/Criteria Provider must submit documentation (such as office chart notes, lab results or other clinical information) supporting that member has met all approval criteria.
It is the policy of health plans affiliated with Centene Corporation® that Nexviazyme is medically necessary when the following criteria are met:
I. Initial Approval Criteria
A. Pompe Disease (must meet all):

1. Diagnosis of late-onset Pompe disease confirmed by one of the following (a or b): a. Enzyme assay confirming low GAA activity; b. DNA testing;
2. Age ≥ 1 year;
   3. Nexviazyme is not prescribed concurrently with Lumizyme®;
   4. Dose does not exceed any of the following (a or b): a. Members weighing ≥ 30 kg: 20 mg/kg every 2 weeks; b. Members weighing < 30 kg: 40 mg/kg every 2 weeks. Approval duration:
      Medicaid/HIM – 6 months Commercial – 6 months or to the member’s renewal date, whichever is longer
      B. Other diagnoses/indications (must meet 1 or 2):

3. If this drug has recently (within the last 6 months) undergone a label change (e.g., newly approved indication, age expansion, new dosing regimen) that is not yet reflected in this policy, refer to one of the following policies (a or b): a. For drugs on the formulary (commercial, health insurance marketplace) or PDL (Medicaid), the no coverage criteria policy for the relevant line of business: CP.CPA.190 for commercial, HIM.PA.33 for health insurance marketplace, and CP.PMN.255 for Medicaid; or Page 1 of 6

   CLINICAL POLICY Avalglucosidase Alfa-ngpt b. For drugs NOT on the formulary (commercial, health insurance marketplace) or PDL (Medicaid), the non-formulary policy for the relevant line of business: CP.CPA.190 for commercial, HIM.PA.103 for health insurance marketplace, and CP.PMN.16 for Medicaid; or

4. If the requested use (e.g., diagnosis, age, dosing regimen) is NOT specifically listed under section III (Diagnoses/Indications for which coverage is NOT authorized) AND criterion 1 above does not apply, refer to the off-label use policy for the relevant line of business: CP.CPA.09 for commercial, HIM.PA.154 for health insurance marketplace, and CP.PMN.53 for Medicaid.
   II. Continued Therapy A. Pompe Disease (must meet all):
5. Member meets one of the following (a or b): a. Currently receiving medication via Centene benefit or member has previously met initial approval criteria; b. Member is currently receiving medication and is enrolled in a state and product with continuity of care regulations (refer to state specific addendums for CC.PHARM.03A and CC.PHARM.03B);
6. Member is responding positively to therapy as evidenced by improvement in the individual member’s Pompe disease manifestation profile (see Appendix D for examples);
7. Nexviazyme is not prescribed concurrently with Lumizyme;
   4. If request is for a dose increase, new dose does not exceed any of the following (a or b): a. Members weighing ≥ 30 kg: 20 mg/kg every 2 weeks; b. Members weighing < 30 kg: 40 mg/kg every 2 weeks. Approval duration: Medicaid/HIM – 12 months Commercial – 6 months or to the member’s renewal date, whichever is longer
      B. Other diagnoses/indications (must meet 1 or 2):
8. If this drug has recently (within the last 6 months) undergone a label change (e.g., newly approved indication, age expansion, new dosing regimen) that is not yet reflected in this policy, refer to one of the following policies (a or b): a. For drugs on the formulary (commercial, health insurance marketplace) or PDL (Medicaid), the no coverage criteria policy for the relevant line of business: CP.CPA.190 for commercial, HIM.PA.33 for health insurance marketplace, and CP.PMN.255 for Medicaid; or b. For drugs NOT on the formulary (commercial, health insurance marketplace) or PDL (Medicaid), the non-formulary policy for the relevant line of business: CP.CPA.190 for commercial, HIM.PA.103 for health insurance marketplace, and CP.PMN.16 for Medicaid; or

9. If the requested use (e.g., diagnosis, age, dosing regimen) is NOT specifically listed under section III (Diagnoses/Indications for which coverage is NOT authorized) AND criterion 1 above does not apply, refer to the off-label use policy for the relevant line Page 2 of 6

   CLINICAL POLICY Avalglucosidase Alfa-ngpt of business: CP.CPA.09 for commercial, HIM.PA.154 for health insurance marketplace, and CP.PMN.53 for Medicaid.
   III. Diagnoses/Indications for which coverage is NOT authorized:
   A. Non-FDA approved indications, which are not addressed in this policy, unless there is sufficient documentation of efficacy and safety according to the off label use policies – CP.CPA.09 for commercial, HIM.PA.154 for health insurance marketplace, and CP.PMN.53 for Medicaid, or evidence of coverage documents. IV. Appendices/General Information Appendix A: Abbreviation/Acronym Key 6MWT: 6 minute walk test FDA: Food and Drug Administration Appendix B: Therapeutic Alternatives
   Not applicable GAA: acid alpha-glucosidase Appendix C: Contraindications/Boxed Warnings • Contraindication(s): none reported • Boxed warning(s): severe hypersensitivity reactions; infusion-associated reactions; risk of acute cardiorespiratory failure in susceptible patients Appendix D: Measures of Therapeutic Response • Pompe disease manifests as a clinical spectrum that varies with respect to age at onset, rate of disease progression, and extent of organ involvement. Patients can present with a variety of signs and symptoms, which can include cardiomegaly, cardiomyopathy, hypotonia, muscle weakness, respiratory distress (eventually requiring assisted ventilation), and skeletal muscle dysfunction. • While there is not one generally applicable set of clinical criteria that can be used to determine appropriateness of continued therapy, clinical parameters that can indicate therapeutic response to Nexviazyme include improved or maintained forced vital capacity, improved or maintained 6 minute walk test (6MWT) distance. Although infantile-onset disease typically presents in the first year of life, age of onset alone does not necessarily distinguish between infantile- and late-onset disease since juvenile-onset disease can present prior to 12 months of age. V. Dosage and Administration
   Indication Dosing Regimen Pompe disease For patients weighing ≥ 30 kg: 20 mg/kg every 2 weeks For patients weighing < 30 kg: 40 mg/kg every 2 weeks Maximum Dose 40 mg/kg/2 weeks VI. Product Availability Lyophilized powder in a single-dose vial: 100 mg Page 3 of 6

   CLINICAL POLICY Avalglucosidase Alfa-ngpt VII.