NEXVIAZYME (avalglucosidase alfa-ngpt)

 NEXVIAZYME (avalglucosidase alfa-ngpt) 

Office Administration - intravenous

Diagnosis considered for coverage:

• Pompe Disease - Indicated for the treatment of patients 1 year of age and older with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency).

Coverage Criteria:

For diagnosis of Pompe disease:

• Dose does not exceed recommended weight-based dosing (see below); AND
  • Less than 30 kg (less than 66 lbs) – 40 mg/kg IV infusion every two weeks
  • 30 kg or more (66 lbs or more) – 20 mg/kg IV infusion every two weeks
• Patient is 1 year of age or older; AND
• Diagnosis of late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) as confirmed by one of the following: AND
  • Deficiency of GAA activity evidenced by a 2% to 40% reduction of normal activity in lymphocytes, fibroblasts, or muscle tissues as confirmed by an enzymatic assay
  • Molecular genetic testing confirms mutations in the GAA gene
• Presence of clinical signs and symptoms of the disease (e.g., respiratory distress, skeletal muscle weakness, etc.).

Reauthorization Criteria:

For diagnosis of Pompe disease:

• Dose does not exceed recommended weight-based dosing; AND
• Documentation of positive clinical response to therapy (e.g., reduced respiratory distress, improved skeletal muscle function, etc.).

Coverage Duration:

• Initial: 1 year
• Reauthorization: 1 year

Authorization is not covered for the following:

• The use of this drug for indications not listed in this policy does not meet the coverage criteria established by the Western Health Advantage (WHA) Pharmacy and Therapeutics (P&T) Committee.

Additional Information:

• Pompe disease (PD) has 2 forms: infantile-onset PD (IOPD), which is considered the classic form, and late-onset PD (LOPD), which can occur in childhood, adolescence, or adulthood. IOPD is considered more severe than LOPD and begins at birth or within the first few months of life. It is characterized by cardiomyopathy and muscle weakness, and it can cause death in the first year of life. The clinical presentation of LOPD differs widely depending on the patient’s specific conditions, resulting in a progressive muscle weakness which is responsible for the motor difficulties and respiratory failure over time.
• Nexviazyme is not approved for the treatment of infantile-onset Pompe disease (IOPD).
• Consensus recommendation based on current clinical guidelines indicate that treatment should be started in patients when they become symptomatic and/or show signs of disease progression.

Policy Updates:

02/15/2022 – New policy approved by P&T.

References:

1. Barba-Romero MA, Barrot E, Bautista-Lorite J, et al. Clinical guidelines for late-onset Pompe disease. Rev Neurol 2012; 54 (8): 497-507.
2. Bay LB, Denzler I, Durand C, et al. Infantile-onset Pompe disease: diagnosis and management. Arch Argent Pediatr. 2019;117(4):271-278.
3. Food and Drug Administration. FDA integrated review for Nexviazyme. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/761194Orig1s000IntegratedR.pdf. August 5, 2021. Accessed December 6, 2021.
4. Kishnani PS, Steiner RD, Bali D, et al. Pompe disease diagnosis and management guideline. Genet Med. May 2006; 8(5): 267–288.
5. Kishnani P, Corzo D, Nicolini M, et al. Recombinant human acid α-glucosidase: Major clinical benefits in infantile-onset Pompe disease. Neurology. 2007;68:99-109.
6. Kronn DF, Salvatore-Day D, Hwu WL, et al. Management of confirmed newborn-screened patients with Pompe disease across the disease spectrum. Pediatrics. 2017;140(s1).e2 0160280.
7. Meena NK, Raben N. Pompe disease: new developments in an old lysosomal storage disorder. Biomolecules. 2020;10:1-19.
8. Nexviazyme [package insert], Cambridge, MA: Genzyme Corporation; August 2021.
9. Schoser B. Pompe disease: what are we missing? Ann Transl Med. 2019;7(13):1-7.
10. Taverna S, Cammarata G, Colomba P, et al. Pompe disease: pathogenesis, molecular genetics and diagnosis. Aging. 2020;12(5):15856-15874.
11. Van der Ploeg AT, Clemens PR, Corzo D et al. A randomized study of alglucosidase alfa in late-onset Pompe disease. N Engl J Med. 2010;362:1396-406.
12. Van der Ploeg AT, Barohn R, Carlson L, et al. Open-label extension study following the Late-Onset Treatment Study (LOTS) of alglucosidase alfa. Mol Genet Metab. 2012;107(3):451-461.