User:Mr. Ibrahem/Olipudase alfa

Olipudase alfa, sold under the brand name Xenpozyme, is a medication used to treat of acid sphingomyelinase deficiency (ASMD), previously known as Niemann-Pick disease type A/B or B. Specifically it is used for non-central nervous system (CNS) symptoms. It is given by gradual injection into a vein.

Common side events include headache, cough, diarrhea, low blood pressure, and itchiness. Other side effects may include infusion reactions, liver problems, and anaphylaxis. Use in pregnancy may harm the baby. It is an enzyme replacement, being manufactured acid sphingomyelinase (ASM), and thereby improves metabolism of fats.

Olipudase alfa was approved for medical use in Japan, Europe, and the United States in 2022. It is also approved in the United Kingdom. In the United States it costs about US$7,500 per 20 mg as of 2022. It is made by recombinant DNA technology.