Pharmacy Prior Authorization Criteria

Xenpozyme (olipudase alfa)

Indications for Prior Authorization

Xenpozyme (olipudase alfa)

• For diagnosis of Acid Sphingomyelinase Deficiency (ASMD)
  Indicated for treatment of non-central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients.

Criteria

Xenpozyme

*Acid Sphingomyelinase Deficiency is also known as Niemann-Pick Disease types A, A/B, and B [1]

Prior Authorization (Initial Authorization)

Length of Approval: 12 Month(s)

• Diagnosis of acid sphingomyelinase deficiency (ASMD)*
AND
• Disease confirmed by ONE of the following: [2]
• Molecular genetic testing confirms biallelic pathogenic variants in the SMPD1 (sphingomyelin phophodiesterase-1) gene
OR
• Residual acid sphingomyelinase activity that is less than 10% of controls (in peripheral blood lymphocytes or cultured skin fibroblasts)
AND
• Submission of medical records (e.g., chart notes) documenting patient has non-central nervous system manifestations of ASMD
AND
• Prescribed by or in consultation with ONE of the following:
• Metabolic disease specialist
• Geneticist

Xenpozyme

Prior Authorization (Reauthorization)

Length of Approval: 24 Month(s)

• Submission of medical records (e.g., chart notes) documenting positive clinical response to therapy (e.g., decrease in spleen size, decrease in liver size, increase in platelet count, improved lung function)

P & T Revisions

2024-10-02, 2023-10-05, 2023-08-30, 2022-11-21

References

1. Healthcare professional brochure. Available at www.xenpozyme.com/pdfs/v0.0.1/hcp/hcp-brochure.pdf. Accessed October 4, 2022.
2. Wasserstein, M., Schuchman, E., et al. Acid Sphingomyelinase Deficiency. Available at https://pubmed.ncbi.nlm.nih.gov/20301544/. Accessed October 4, 2022.
3. McGovern, M., Dionisi-Vici, C., et al. Consensus recommendation for a diagnostic guideline for acid sphingomyelinase deficiency. Available at https://pubmed.ncbi.nlm.nih.gov/28406489/. Accessed October 4, 2022.
4. Living with ASMD. Available at Proactive Symptom Management While Living with ASMD (asmdfacts.com). Accessed October 4, 2022.
5. Xenpozyme prescribing information. Cambridge, MA. Genzyme Corporation. August 2022.

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Revision History

• 2024-10-02: 2024 Annual Review.
• 2023-10-05: 2023 Annual Review
• 2023-08-30: update guideline
• 2022-11-21: New UM PA criteria

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