Medical Drug Criteria (MDC)

Policy Num:       P1.001.015
Policy Name:     XENPOZYME^® (olipudase alfa-rpcp)
Policy ID:          [P1.001.015] [Ac/L/ M+/ P +] [0.00.00]

Last Review:       September 17, 2024
Next Review:      September 20, 2025
 

Related MDC: NONE

XENPOZYME^® (olipudase alfa-rpcp) 

Summary

Olipudase alfa (Xenpozyme) is an enzyme replacement therapy, for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients. Acid sphingomyelinase deficiency (ASMD) is an autosomal recessive lysosomal storage disorder caused by a mutation on the sphingomyelin phophodiesterase-1 (SMPD1) gene. It is characterized by accumulation of undegraded sphingomyelin in the spleen, liver, lungs, bone marrow, and brain due to a deficiency or insufficient activity of the enzyme ASM. Symptoms may include lack of muscle coordination, brain degeneration, learning problems, loss of muscle tone, increased sensitivity to touch, spasticity, feeding and swallowing difficulties, slurred speech, and an enlarged liver and spleen. ASMD is also known as Niemann-Pick disease (NPD) types A and B. NPD type C is now considered a separate disorder, distinct from NPD types A and B. 

Policy Statements

Xenopozyme may be considered medically necessary in adult and pediatric patients for the treatment of non-central nervous system (non-CNS) manifestations of ASMD. 

Policy Guidelines

Coverage eligibility for Olipudase alfa (Xenpozyme) will be considered when the following criteria are met:

•    Authorization of 12 months may be granted for treatment of adult and pediatric patients with non-central nervous system (non-CNS) manifestations of ASMD

DOSAGE/ADMINISTRATION

For treatment of non-central nervous system (non-CNS) manifestations of ASMD): 

•    Adults: Recommended starting dose is 0.1 mg/kg administered as an intravenous infusion, titrate every 2 weeks to 3 mg/kg.

•    Pediatrics: Recommended starting dose is 0.03 mg/kg administered as an intravenous infusion, titrate every 2 weeks to 3 mg/kg.
 

REQUIRED MEDICAL INFORMATION

Initial Therapy

a) Initiation of olipudase alfa (Xenpozyme) meets the definition of medical necessity when ALL of the following criteria are met: 

1.    Member is diagnosed with acid sphingomyelinase deficiency (ASMD)

2.    Member has a mutation in the sphingomyelin phophodiesterase-1 (SMPD1) gene (laboratory documentation must be provided)

3.    Member has a deficiency of acid sphingomyelinase as measured in peripheral leukocytes, cultured fibroblasts, or lymphocytes – laboratory documentation must be provided 

4.    Member has a spleen volume greater than or equal to 5 multiples of normal (MN) measured by magnetic resonance imaging (MRI) 

5.    Member has a diffuse capacity of the lung for carbon monoxide less than or equal to 70% of the predicted normal value 

6.    Olipudase is prescribed by specializing in the treatment of ASMD (e.g., neurologist, hepatologist, geneticist)

Renewal Approval Criteria 

a)    Continuation of olipudase alfa (Xenpozyme) meets the definition of medical necessity when ALL of the following criteria are met: 

1.    The member has previously met all indication-specific initial criteria 

2.    Olipudase is prescribed by a provider specializing in the treatment of ASMD (e.g., neurologist, hepatologist, geneticist) 

3.    Dose does not exceed 3 mg/kg every 2 weeks
 

EXCLUSION CRITERIA

NONE

BENEFIT APPLICATION

Benefits are determined by the group contract, member benefit booklet, and/or individual subscriber certificate in effect at the time services were rendered. Benefit products or negotiated coverages may have all or some of the services discussed in this medical policy excluded from their coverag. 

OTHER CRITERIA

NONE 

Population Reference No. 1 Policy Statement

References

1. Clinical Pharmacology [Internet]. Tampa (FL): Gold Standard, Inc.; 2022 [cited 9/20/22]. Available from: http://www.clinicalpharmacology.com/. 
2. ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine; 2000 Feb 29 - [cited 9/20/22]. Available from: http://clinicaltrials.gov/. 
3. Cox GF, et al. Burden of illness in acid sphingomyelinase deficiency: A retrospective chart review of 100 patients. JIMD Rep. 2018; 41:119-129. doi:10.1007/89042018120. 
4. DRUGDEX® System [Internet]. Greenwood Village (CO): Thomson Micromedex; Updated periodically [cited 9/20/23]. 
5. National Organization for Rare Disorders. Acid sphingomyelinase deficiency. 2022. https://rarediseases.org/rare-diseases/acid-sphingomyelinase-deficiency/. 
6. Xenpozyme [package insert]. Cambridge, MA; Genzyme Corporation, Inc.; July 2023. Accessed  Revised: 12/2023

Codes

Applicable Modifiers

N/A

Policy History

Payment Policy Guidelines

Administrative Evaluation

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Economic Impact

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