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What is Olipudase Alfa used for?

An enzyme replacement therapy for a rare inherited disease called acid sphingomyelinase deficiency, in which harmful lipids accumulate in the liver, spleen, and lungs. Regular infusions help break down these accumulated substances and improve organ function.

How does Olipudase Alfa work?

Provides exogenous replacement of the deficient or absent enzyme, enabling the metabolic processing of accumulated substrates and restoring normal metabolic function.

Is Olipudase Alfa an approved drug?

Yes, Olipudase Alfa is an approved drug. It has reached clinical phase 4. It is classified as a Enzyme.

{# References & Data Sources section for drug detail pages. Renders standard pharmacological database links plus the drug's data_sources field. #}

References & Data Sources

• ChEMBL — European Bioinformatics Institute (EBI). CHEMBL3707358. Open-access bioactivity database.

Data aggregated from publicly available pharmacological databases. Last updated 2026-02-27.