Pharmacy Prior Authorization Criteria

Lumizyme (alglucosidase alfa)

Indications for Prior Authorization

Lumizyme (alglucosidase alfa)

• For diagnosis of Pompe Disease
  Indicated for patients with Pompe disease [acid alpha-glucosidase (GAA) deficiency].

Criteria

Lumizyme

Prior Authorization (Initial Authorization)

Length of Approval: 12 Month(s)
For diagnosis of Infantile Onset Pompe Disease (IOPD)

• Diagnosis of infantile-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) as confirmed by one of the following: [3]
• Absence or deficiency (less than 1% of the lab specific normal mean) of GAA enzyme activity in lymphocytes, fibroblasts, or muscle tissues as confirmed by an enzymatic assay
OR
• Molecular genetic testing confirms mutations in the GAA gene
AND
• Presence of clinical signs and symptoms of the disease (e.g., cardiomegaly, hypotonia, etc.)
AND
• Patient is less than or equal to 12 months of age

Lumizyme

Prior Authorization (Reauthorization)

Length of Approval: 24 Month(s)
For diagnosis of Infantile Onset Pompe Disease (IOPD)

• Patient demonstrates positive clinical response to therapy

Lumizyme

Prior Authorization (Initial Authorization)

Length of Approval: 12 Month(s)
For diagnosis of Late Onset Pompe Disease (LOPD)

• Diagnosis of late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) as confirmed by one of the following: [3, 5]
• Absence or deficiency (less than 40% of the lab specific normal mean) of GAA enzyme activity in lymphocytes, fibroblasts, or muscle tissues as confirmed by an enzymatic assay
OR
• Molecular genetic testing confirms mutations in the GAA gene
AND
• Presence of clinical signs and symptoms of the disease (e.g., respiratory distress, skeletal muscle weakness, etc.) [A]
AND
• Patient is 1 year of age or older

Lumizyme

Prior Authorization (Reauthorization)

Length of Approval: 24 Month(s)
For diagnosis of Late Onset Pompe Disease (LOPD)

• Patient demonstrates positive clinical response to therapy

P & T Revisions

2024-05-03, 2023-10-03, 2023-05-05, 2022-05-03, 2021-11-04, 2021-09-27, 2021-05-20

References

1. Lumizyme Prescribing Information. Genzyme Corporation. Cambridge, MA. March 2024.
2. Kronn DF, Day-Salvatore D, Hwu WL, et al. Management of Confirmed Newborn- Screened Patients With Pompe Disease Across the Disease Spectrum.
3. Kishani PS, Steiner RD, Bali, D. ACMG Practice Guideline. Pompe disease diagnosis and management guideline. Genet Med. 2006;8(5):267-88.
4. Diagnosing Pompe Disease (also known as Acid Maltase Deficiency). Available at: https://www.pompe.com/-/media/EMS/Conditions/RareDiseases/Brands/pompe-us/hcp/PDF/SAUSPD18042050bk1vFinal10.pdf?la=en-US and https://www.pompe.com/-/media/EMS/Conditions/RareDiseases/Brands/pompe-us/hcp/PDF/SAUSPD18042050bj1vFinal10.pdf?la=en-US. Accessed May 12, 2020.
5. Barba-Romero MA, Barrot E, Bautista-Lorite J, et al. Clinical guidelines for late-onset Pompe disease. Rev Neurol 2012; 54 (8): 497-507.

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End Notes

1. Consensus recommendation based on current clinical guidelines indicate that treatment should be started in patients with late onset Pompe disease when they become symptomatic and/or show signs of disease progression [3, 5].

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Revision History

• 2024-05-03: Annual review: No criteria changes. Updated references.
• 2023-10-03: Program update to standard reauthorization language. No changes to clinical intent
• 2023-05-05: Annual review: No criteria changes. Updated reauthorization criteria approval length to 24 months for both indications. Updated references.
• 2022-05-03: Annual review: No criteria changes.
• 2021-11-04: updated criteria, background, references
• 2021-09-27: Annual review
• 2021-05-20: Annual review

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