XENPOZYME (olipudase alfa-rpcp)

Medical Administration – intravenous

Diagnosis considered for coverage:
  • Indicated for treatment of non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients
Coverage Criteria:

For diagnosis of ASMD:

  • Dose does not exceed 3 mg/kg intravenous infusion every 2 weeks; AND
  • Documented diagnosis of acid sphingomyelinase deficiency (ASMD); AND
  • Prescribed by or in consultation with a metabolic disease specialist or geneticist; AND
  • Disease confirmed by ONE of the following:
    • Molecular genetic testing confirms biallelic pathogenic variants in the SMPD1 (sphingomyelin phophodiesterase-1) gene
    • Residual acid sphingomyelinase activity that is less than 10% of controls (in peripheral blood lymphocytes or cultured skin fibroblasts); AND
  • Medical records (e.g., chart notes) document patient has non-central nervous system manifestations of ASMD; AND
  • Patient has ASMD Type B or Type A/B; AND
  • Documentation of patient’s current weight
Reauthorization Criteria:

For diagnosis of ASMD:

  • Dose does not exceed 3 mg/kg intravenous infusion every 2 weeks; AND
  • 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); AND
  • Documentation of patient’s current weight
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: 
  • Recommended starting dose:
    • Pediatric patients: 0.03 mg/kg intravenous infusion every 2 weeks
    • Adults: 0.1 mg/kg intravenous infusion every 2 weeks
  • Recommended maintenance dose:
    • Pediatric patients: 3 mg/kg intravenous infusion every 2 weeks
    • Adults: 3 mg/kg intravenous infusion every 2 weeks
  • Non-central nervous system manifestations of ASMD: hepatosplenomegaly, interstitial lung disease, bone marrow infiltration, etc
  • The safety and effectiveness of XENPOZYME for the treatment of non-central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) have been established in pediatric patients down to birth
Policy Updates:
  • 3/1/2023 – New policy approved by P&T
References:
  • Healthcare professional brochure. Available at www.xenpozyme.com/pdfs/v0.0.1/hcp/hcp-brochure.pdf. Accessed October 4, 2022. 
  • Wasserstein, M., Schuchman, E., et al. Acid Sphingomyelinase Deficiency. Available at https://pubmed.ncbi.nlm.nih.gov/20301544/. Accessed October 4, 2022. 
  • 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. 
  • Living with ASMD. Available at Proactive Symptom Management While Living with ASMD (asmdfacts.com). Accessed October 4, 2022. 
  • Xenpozyme prescribing information. Cambridge, MA. Genzyme Corporation. August 2022. 

Last review date: March 1, 2023

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