Xenpozyme™ (olipudase alfa-rpcp intravenous infusion)

Covered: Xenpozyme (olipudase alfa) is authorized for non‑CNS manifestations of ASMD in adults and pediatric patients (limited to ASMD type B or A/B) and excludes CNS disease, ASMD type A, and any patient without documented biallelic SMPD1 pathogenic variants or where Gaucher disease has not been excluded. Key requirements: approval requires enzymatic assay showing deficient ASM activity and genetic confirmation of biallelic SMPD1 variants, documentation of ≥2 non‑CNS ASMD signs, prescribing by or in consultation with a geneticist/endocrinologist/metabolic or lysosomal storage disorder specialist, adherence to the specified dose‑escalation and BMI‑based dosing (adjusted weight if BMI>30), and authorization is limited to 12 months.

"FDA-approved indication: Treatment of non-central nervous system (CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients."