An Intracellular Enzyme Drug via Endocytosis: Olipudase Alfa (Xenpozyme)

In August 2022, olipudase alfa (Xenpozyme), a hydrolytic lysosomal sphingomyelin-specific enzyme indicated for non–CNS manifestations of acid sphingomyelinase deficiency (ASMD), was approved in the US and Europe.

This Premium deep dive into Xenpozyme includes:

  • the industry context,
  • the clinical data,
  • the target rationale,
  • the mechanism of action,
  • and the drug’s origins.

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