Xenpozyme™

Drug - Xenpozyme™ (olipudase alfa-rpcp) [Genzyme Corporation]

March 2025

Therapeutic Area - Acid sphingomyelinase deficiency (ASMD)

Initial approval criteria

Patients of reproductive potential will have pregnancy status verified prior to start of therapy and will use effective contraception during treatment and for 14 days after the last dose of therapy is discontinued; AND
Patient has documented baseline measures (necessary for renewal) of: percent predicted diffusion capacity of the lungs for carbon monoxide (DLco) or other age-appropriate pulmonary function testing, spleen volume, liver volume, plasma lyso-sphingomyelin, and/or platelet count (height Z-score and skeletal maturation are relevant for pediatric patients); AND

Universal criteria

Documented baseline transaminase (alanine aminotransferase [ALT] and aspartate aminotransferase [AST]) levels within 1 month prior to treatment initiation, within 72 hours prior to any infusion during dose escalation, and periodically throughout therapy; AND
Patient should not require invasive ventilatory support OR non-invasive ventilatory support while awake and for >12 hours a day; AND

Acid Sphingomyelinase Deficiency (ASMD) (Niemann-Pick Disease):

Patient has a definitive diagnosis of ASMD as confirmed by the following:
Detection of biallelic pathogenic mutations in the SMPD1 gene by molecular genetic testing; OR
Deficiency of acid sphingomyelinase enzyme activity <10% of controls as measured in peripheral leukocytes, cultured fibroblasts, or lymphocytes; AND
Patient has a clinical diagnosis consistent with Niemann-Pick disease type B (NPD-B) or A/B (NPD-A/B); AND
Therapy will be used for non-CNS manifestations of disease

Patient continues to meet universal and other indication-specific relevant criteria such as concomitant therapy requirements (not including prerequisite therapy), performance status, etc. identified in the initial approval criteria; AND
Absence of unacceptable toxicity from the drug. Examples of unacceptable toxicity include anaphylaxis and severe hypersensitivity reactions, severe infusion-associated reactions, severely elevated liver transaminases, etc.; AND
Patient has not experienced progressive/irreversible severe cognitive impairment; AND
Disease response with treatment as defined by improvement or stability from pre-treatment baseline by the following:
- Improvement in or stability in the percent predicted diffusion capacity of the lungs for carbon monoxide (DLco) or other age-appropriate pulmonary function testing, OR
- Improvement in or stability of spleen and/or liver volumes; OR
- Reduction in plasma lyso-sphingomyelin; OR
- Improvement in or stability of platelet count; OR
- Improvement in linear growth progression as measured by mean height Z-scores (pediatric patients only)

3mg/kg every 2 weeks
Patient’s patient’s body mass index (BMI) AND body weight (in kg) must be submitted at time of request:
If patient’s BMI is less than or equal to 30, submit patient’s actual body weight (kg)
If patient’s BMI is greater than 30, submit patient’s adjusted body weight (kg)

Xenpozyme must be billed as a medical claim.

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