The following table summarises the key steps and dates for this application.
|Designation, Orphan||13 November 2019|
|Submission dossier accepted and first round evaluation commenced||29 January 2020|
|First round evaluation completed||30 June 2020|
|Sponsor provides responses on questions raised in first round evaluation||31 August 2020|
|Second round evaluation completed||19 October 2020|
|Delegate's overall benefit-risk assessment and request for Advisory Committee advice||29 October 2020|
|Sponsor's pre-Advisory Committee response||11 November 2020|
|Advisory Committee meeting|| |
3 and 4 December 2020
4 and 5 February 2021
|Registration decision (Outcome)||24 February 2021|
|Completion of administrative activities and registration on ARTG||4 March 2021|
|Number of working days from submission dossier acceptance to registration decision*||199|
*Statutory timeframe for standard applications is 255 working days
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.
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.
- 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.