How does Xenpozyme work?

Drugs.com

Official answer

by Drugs.com

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.

Related medical questions

Disclaimer

Every effort has been made to ensure that the information provided by Drugslib.com is accurate, up-to-date, and complete, but no guarantee is made to that effect. Drug information contained herein may be time sensitive. Drugslib.com information has been compiled for use by healthcare practitioners and consumers in the United States and therefore Drugslib.com does not warrant that uses outside of the United States are appropriate, unless specifically indicated otherwise. Drugslib.com's drug information does not endorse drugs, diagnose patients or recommend therapy. Drugslib.com's drug information is an informational resource designed to assist licensed healthcare practitioners in caring for their patients and/or to serve consumers viewing this service as a supplement to, and not a substitute for, the expertise, skill, knowledge and judgment of healthcare practitioners.

The absence of a warning for a given drug or drug combination in no way should be construed to indicate that the drug or drug combination is safe, effective or appropriate for any given patient. Drugslib.com does not assume any responsibility for any aspect of healthcare administered with the aid of information Drugslib.com provides. The information contained herein is not intended to cover all possible uses, directions, precautions, warnings, drug interactions, allergic reactions, or adverse effects. If you have questions about the drugs you are taking, check with your doctor, nurse or pharmacist.

Popular Keywords