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 (olipudase alfa) is the first approved enzyme replacement therapy for acid sphingomyelinase deficiency (ASMD), a rare progressive genetic disorder. Approved by the FDA and EMA, it targets the underlying enzyme deficiency to reduce lipid buildup in visceral organs. Eligible patients can access this treatment in the Boao Lecheng pilot zone under special import policies.

Regulatory Approvals

• FDA — approved (2022) — label
• EMA — approved (2022) — source

Pivotal Clinical Evidence

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

Sources

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