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Zolgensma

10 March 2021

The Australian Prescription Medicine Decision Summary provides a short overview of the TGA's evaluation process leading to the registration of a new prescription medicine on the Australian Register of Therapeutic Goods (ARTG).

More in-depth information about the evaluation will be available in the Australian Public Assessment Report (AusPAR) for a particular prescription medicine, which can be found on the AusPAR search page once published.

Australian prescription medicine decision summary

Summary of submission

Submission type
New biological entity
Product name

Zolgensma

Active ingredients
Onasemnogene abeparvovec
ATC codes
M09AX09
Decision
Approved
Date of decision
24 February 2021
Date of entry onto ARTG
4 March 2021
Original publication date
10 March 2021
ARTG numbers
327905, 327906, 327907
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.
Sponsor
Novartis Pharmaceuticals Australia Pty Limited
Sponsor address
54 Waterloo Road, Macquarie Park NSW 2113
Dose forms
Injection for intravenous infusion
Strength
2 x 1013 vector genomes/mL
Other ingredients
Trometamol, magnesium chloride hexahydrate, sodium chloride, poloxamer, hydrochloric acid (for pH adjustment) and water for injections
Containers
Vial (two fill volumes: 5.5 mL or 8.3 mL)
Pack sizes

The dose of Zolgensma and exact number of vials required for each patient is calculated according to the patient's weight (refer to the Product Information for further information). The pack sizes are:

  • Packs consisting of two to nine of the 8.3 mL vials, and
  • A composite pack consisting of various combinations of the 5.5 mL and 8.3 mL vials (refer to the Product Information for further information).

Note: the 5.5 mL vial is not supplied on its own.

Routes of administration
Intravenous infusion
Dosage

For single-dose intravenous infusion only.

Treatment with Zolgensma should be supervised by a physician experienced in the management of patients with spinal muscular atrophy (SMA).

In order to improve the traceability of biological medicinal products, the name and the batch number of the administered product should be clearly recorded.

An immune response to the adeno-associated viral vector serotype 9 (AAV9) capsid will occur after infusion of Zolgensma, thus patients should not be re-dosed with Zolgensma.

Zolgensma is for a single treatment only.

The recommended dose of Zolgensma is 1.1 × 1014 vector genomes per kilogram (vg/kg) of body weight (see Table 1 in the Product Information).

For further information on dosage, refer to the Product Information.

Pregnancy category

B2

Drugs which have been taken by only a limited number of pregnant women and women of childbearing age, without an increase in the frequency of malformation or other direct or indirect harmful effects on the human fetus having been observed.

Studies in animals are inadequate or may be lacking, but available data show no evidence of an increased occurrence of fetal damage.

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?

Zolgensma (onasemnogene abeparvovec) was approved for the following therapeutic use:

Zolgensma (onasemnogene abeparvovec) is indicated for the treatment of paediatric patients less than 9 months of age with symptomatic or pre-symptomatic spinal muscular atrophy with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene and 1 to 3 copies of the SMN2 gene.

What is this medicine and how does it work?

What was the decision based on?

What steps were involved in the decision process?

What post-market commitments will the sponsor undertake?

  • Zolgensma (onasemnogene abeparvovec) is to be included in the Black Triangle Scheme. The Product Information (PI) and Consumer Medicines Information (CMI) for Zolgensma 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 Zolgensma European Union (EU)-Risk Management Plan (RMP), (version 0.7, dated 19 March 2020; data lock point 31 December 2019) with Australian Specific Annex (version 1.1, dated 17 August 2020), included with submission PM-2019-05979-1-3, 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 (Rev 1), Part VII.B Structures and processes. Note that submission of a PSUR does not constitute an application to vary the registration.

  • Preparation and administration of Zolgensma is restricted to clinical facilities accredited to the National Safety and Quality Health Service (NSQHS) Standards.
  • Laboratory testing and compliance with Certified Product Details (CPD)
    • All batches of Zolgensma supplied in Australia must comply with the product details and specifications approved during evaluation and detailed in the Certified Product Details (CPD).
    • 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.

  • For all injectable products the Product Information must be included with the product as a package insert.

Further information

The latest Product Information (PI) and Consumer Medicines Information (CMI) can be found at: ARTG search.

Australian Public Assessment Reports (AusPARs) can be found at: AusPAR search.

The latest news and updates regarding therapeutic goods regulation can be found at: TGA news room.