WHA
 

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

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]
    • Presence of biallelic pathogenic variants in the SMPD1 (sphingomyelin phophodiesterase-1) gene as detected by an FDA-approved test or a test performed at a facility approved by Clinical Laboratory Improvement Amendments (CLIA)
      • 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)

  • Guideline ID
    GL-442216

  • Formulary
    Premium

  • Effective date
    Feb 1, 2026

  • P&T approval date
    Nov 17, 2022

P & T Revisions

2025-12-02, 2024-10-02, 2023-10-05, 2023-08-30, 2022-11-21

  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. December 2024.
  • 2025-12-02: 2025 Annual Review. Update to standard genetic testing language, from “Molecular genetic testing confirms” to “Presence of biallelic pathogenic variants in the SMPD1 (sphingomyelin phophodiesterase-1) gene as detected by an FDA-approved test or a test performed at a facility approved by Clinical Laboratory Improvement Amendments (CLIA).” Update reference.
  • 2024-10-02: 2024 Annual Review.
  • 2023-10-05: 2023 Annual Review
  • 2023-08-30: update guideline
  • 2022-11-21: New UM PA criteria