Pharmacy Prior Authorization Criteria

Pombiliti (cipaglucosidase alfa-atga)

Indications for Prior Authorization

Pombiliti (cipaglucosidase alfa-atga)

• For diagnosis of Late-Onset Pompe disease
  Indicated, in combination with Opfolda, an enzyme stabilizer, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT).

Criteria

Pombiliti

Prior Authorization (Initial Authorization)

Length of Approval: 12 Month(s)

• Diagnosis of late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency)
AND
• Disease is confirmed by one of the following: [2, 4-5]
• 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
• 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
• Medication is used in combination with Opfolda (miglustat)
AND
• Patient weight is greater than or equal to 40 kg
AND
• Trial and inadequate response to one of the following:
• Lumizyme
• Nexviazyme
AND
• Not to be used in combination with other miglustat products (i.e., Zavesca, Yargesa)

Pombiliti

Prior Authorization (Reauthorization)

Length of Approval: 24 Month(s)

• Patient demonstrates positive clinical response to therapy (e.g., improvement in FVC, improvement in 6-minute walk distance [6MWD])
AND
• Medication is used in combination with Opfolda (miglustat)
AND
• Not to be used in combination with other miglustat products (i.e., Zavesca, Yargesa)

P & T Revisions

2023-11-26

References

1. Pombiliti Prescribing Information. Amicus Therapeutics US, LLC. Philadelphia, PA. Sept 2023.
2. Diaz, C., Diaz-Manera, J. Therapeutic Options for the Management of Pompe Disease: Current Challenges and Clinical Evidence in Therapeutics and Clinical Risk Management. Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9759116/. Accessed November 2, 2023.
3. Cleveland Clinic - Pompe Disease. Available at: https://my.clevelandclinic.org/health/diseases/15808-pompe-disease. Accessed November 2, 2023.
4. Cupler, E., Berger, K., Leshner, R., et al. Consensus Treatment Recommendations for Late-Onset Pompe Disease. Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3534745/. Accessed November 2, 2023.
5. Barba-Romero MA, Barrot E, Bautista-Lorite J, et al. Clinical guidelines for late-onset Pompe disease. Available at: https://www.orpha.net/data/patho/Cpg/en/PompeLateOnset_ES_en_CPG_ORPHA420429.pdf. Accessed November 2, 2023.

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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 [2, 4-5].

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Revision History

• 2023-11-26: New UM PA criteria

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