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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