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Xenpozyme
Published
Product name
Xenpozyme
Active ingredient
Olipudase alfa
Submission type
New Entity
Decision
Approved
Decision date
Registration date
What this medicine was approved for
Xenpozyme (olipudase alfa) was approved for the following therapeutic use:
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 (Niemann-Pick type A/B) or type B (Niemann-Pick type B).
How this medicine works
Olipudase alfa is a recombinant human acid sphingomyelinase that reduces sphingomyelin (SM) accumulation in organs of patients with acid sphingomyelinase deficiency (ASMD).
Why the TGA approved or did not approve this medicine
The decision was based on quality (chemistry and manufacturing), nonclinical (pharmacology and toxicology), clinical (pharmacology, safety and efficacy) and risk management plan information submitted by the sponsor. The benefit-risk profile of Xenpozyme was considered favourable for the therapeutic use approved.
Sponsor