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Device/Product name
Active Ingredient
Olipudase alfa
Date of decision
Submission type
New Entity
ATC codes
What was the decision based on
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.
What steps were involved in the decision process

Registration timeline

The following table summarises the key steps and dates for this comparable overseas regulator approach A (COR-A) application.



Designation (Orphan)

21 June 2022

Submission dossier accepted and first round evaluation commenced

31 October 2022

Delegate’s Overall benefit-risk assessment and request for Advisory Committee advice

28 February 2023

Advisory Committee meeting

30 and 31 March 2023

Registration decision (Outcome)

21 August 2023

Completion of administrative activities and registration on ARTG

24 August 2023

Number of working days from submission dossier acceptance to registration decision*


* The COR-A process has a 120 working day evaluation and decision timeframe.

Date of entry onto ARTG
Black triangle scheme
Yes. This product will remain in the scheme for 5 years, starting on the date the product is first supplied in Australia
Dose forms
Powder for injection
20 mg
Pack sizes
1, 5, 10, 25
Routes of administration
Intravenous infusion

Xenpozyme treatment should be initiated and supervised by a physician experienced in the management of ASMD or other inherited metabolic disorders.

Xenpozyme infusion should be administered by a healthcare professional with access to appropriate medical support to manage potential severe reactions, including serious systemic hypersensitivity reactions and anaphylaxis. Xenpozyme dose regimen is dependent on the age and weight of patient.  Treatment with Xenpozyme should always be initiated via a dose escalation regimen followed by a maintenance dose. The dose escalation regimens are different for adult and paediatric patients.

For further information refer to the Product Information.

Pregnancy category

Drugs which have caused, are suspected to have caused or may be expected to cause, an increased incidence of human fetal malformations or irreversible damage. These drugs may also have adverse pharmacological effects. Accompanying texts should be consulted for further details.

The use of any medicine during pregnancy requires careful consideration of both risks and benefits by the treating health professional. This must not be used as the sole basis of decision making in the use of medicines during pregnancy. The TGA does not provide advice on the use of medicines in pregnancy for specific cases. More information is available from obstetric drug information services in your State or Territory.
What was approved

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).

What is this medicine and how does it work
Olipudase alfa is a recombinant human acid sphingomyelinase that reduces sphingomyelin (SM) accumulation in organs of patients with acid sphingomyelinase deficiency (ASMD).
What post-market commitments will the sponsor undertake
  • Xenpozyme (olipudase alfa) is to be included in the Black Triangle Scheme. The PI [Product Information] and CMI [Consumer Medicines Information] for Xenpozyme must include the black triangle symbol and mandatory accompanying text for five years, which starts from the date that the sponsor notifies the TGA of supply of the product.

  • The Xenpozyme EU [European Union]-risk management plan (RMP) (version 2.2, dated 2 March 2023, data lock point 31 October 2022), with Australia specific annex (version 2.1, dated May 2023), included with Submission PM-2022-03512-1-3, and any subsequent revisions, as agreed with the TGA will be implemented in Australia.

An obligatory component of risk management plans is routine pharmacovigilance. Routine pharmacovigilance includes the submission of periodic safety update reports (PSURs).

Reports are to be provided in line with the current published list of EU reference dates and frequency of submission of PSURs until the period covered by such reports is not less than three years from the date of the approval letter.

The reports are to at least meet the requirements for PSURs as described in the European Medicines Agency’s Guideline on Good Pharmacovigilance Practices (GVP) Module VII-periodic safety update report (revision 1), Part VII.B Structures and processes. Note that submission of a PSUR does not constitute an application to vary the registration. Each report must have been prepared within ninety calendar days of the data lock point for that report.

  • Clinical condition

Provision of the final clinical study report of the open-label extension study, using the appropriate application type.

  • Quality conditions

Laboratory testing & compliance with Certified Product Details (CPD)

  1. All batches of Xenpozyme olipudase alfa supplied in Australia must comply with the product details and specifications approved during evaluation and detailed in the Certified Product Details (CPD).
  2. When requested by the TGA, the Sponsor should be prepared to provide product samples, specified reference materials and documentary evidence to enable the TGA to conduct laboratory testing on the Product. Outcomes of laboratory testing are published biannually in the TGA Database of Laboratory Testing Results and periodically in testing reports on the TGA website.

  • Certified Product Details

The Certified Product Details (CPD), as described in Guidance 7: Certified Product Details of the Australian Regulatory Guidelines for Prescription Medicines (ARGPM), in PDF format, for the above products should be provided upon registration of these therapeutic goods. In addition, an updated CPD should be provided when changes to finished product specifications and test methods are approved in a Category 3 application or notified through a self-assessable change. A template for preparation of CPD for biological prescription medicines can be obtained from the TGA website:

[for the form]

[for the CPD guidance]

Further information

The latest Product Information (PI) and Consumer Medicines Information (CMI) can be found by Searching the Australian Register of Therapeutic Goods (ARTG).

Australian Public Assessment Reports (AusPARs) can be found by searching our AusPAR dataset.

The latest news and updates regarding therapeutic goods regulation can be found on our news page.

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