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.