XENPOZYME (olipudase alfa-rpcp)

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.