XENPOZYME olipudase alfa

Drug Class

Recombinant human acid sphingomyelinase (rhASM) enzyme replacement therapy

Mechanism

Replaces deficient acid sphingomyelinase (ASM), an enzyme that catalyzes the hydrolysis of sphingomyelin to ceramide and phosphorylcholine, preventing lipid accumulation in tissues.

Approved Indication

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

What This Means For Patients

XENPOZYME is not distributed through mainland China's retail pharmacy network. Uwell Cloud can help arrange a purchase channel for this medicine, sourced and dispatched from Hong Kong; clinical administration support in China can be coordinated on request. Availability and eligibility for any specific patient are determined by clinical review.

Regulatory Approvals

• FDA — Approved (2022) — Sources
• EMA — Approved (2022) — Sources

Pivotal Clinical Evidence

• ASCEND — Wasserstein et al., Genet Med 2022 (NCT02004704) — Sources

Sources

• https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/761234s000lbl.pdf
• https://www.ema.europa.eu/en/medicines/human/EPAR/xenpozyme