Xenpozyme is a medicine for treating patients with acid sphingomyelinase deficiency (ASMD), a genetic condition, historically known as Niemann\-Pick disease type A, A/B and B. There are three types of Niemann\-Pick disease (A, B and C), with different genetic causes and different symptoms. Xenpozyme is used to treat patients with type A/B or type B. It is intended to treat the symptoms of ASMD that are not related to the brain. Niemann\-Pick disease is rare, and Xenpozyme was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 5 December 2016\. Further information on the orphan designation can be found here: ema.europa.eu/medicines/human/orphan\-designations/eu\-3\-01\-056\. Xenpozyme contains the active substance olipudase alfa.
Therapeutic Indication
Xenpozyme is indicated as an enzyme replacement therapy for the treatment of non\-Central Nervous System (CNS) manifestations of Acid Sphingomyelinase Deficiency (ASMD) in paediatric and adult patients with type A/B or type B.
Therapeutic Area (MeSH)
ATC Code
A16AB25
ATC Item
N/A
Pharmacotherapeutic Group
Other alimentary tract and metabolism products
Active Substance (Summary)
INN / Common Names
EMA Name
Xenpozyme
Medicine Name
Xenpozyme
Aliases
N/ANo risk management plan link.
