How does Xenpozyme work?

Xenpozyme (olipudase alfa-rpcp) is an enzyme replacement therapy that works by providing a functional man-made enzyme to replace acid sphingomyelinase (ASM), an enzyme lacking in people with ASM deficiency (ASMD).

ASMD is a rare genetic condition (previously known as Niemann-Pick disease types A, A/B and B), which causes damage to multiple organs in the body including the liver, lungs, heart and spleen. It can also impact the digestive system and blood. It is a type of lysosomal storage disease.

ASMD is caused by a mutation in the sphingomyelin phophodiesterase-1 (SMPD1) gene, which results in a deficiency in the number of functional copies of the ASM enzyme. ASM is necessary for the breakdown of a lipid (fatty substance) known as sphingomyelin, which plays an important role in maintaining the structure of cell membranes. If sphingomyelin isn’t broken down due to a deficiency of ASM it accumulates and causes damage.

Does Xenpozyme cure acid sphingomyelinase deficiency (ASMD)?

Xenpozyme does not cure ASMD, but simply replaces the ASM enzyme that people with ASMD are missing. Xenpozyme needs to be taken on an ongoing basis and is administered via intravenous (IV) infusion every two weeks.