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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
|Submission type|| |
New biological entity
|Product name|| |
|Active ingredients|| |
|ATC codes|| |
|Date of decision|| |
4 August 2020
|Date of entry onto ARTG|| |
5 August 2020
|Original publication date|| |
13 August 2020
|ARTG numbers|| |
|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
Novartis Pharmaceuticals Australia Pty Ltd
|Sponsor address|| |
54 Waterloo Road, Macquarie Park NSW 2113
|Dose forms|| |
Concentrated solution for subretinal injection, with diluent
5 x 1012 vector genomes (vg) / mL
|Other ingredients|| |
For both concentrate and diluent: sodium chloride, monobasic sodium phosphate, dibasic sodium phosphate, poloxalene, water
|Pack sizes|| |
1 vial of concentrate with 2 vials of diluent
|Routes of administration|| |
Treatment should be initiated and administered by a retinal surgeon experienced in performing macular surgery.
Patients will receive a single dose of 1.5 x 1011 vg of Luxturna in each eye. Each dose will be delivered into the subretinal space in a total volume of 0.3 mL. The individual administration procedure to each eye is performed on separate days within a close interval, but no fewer than 6 days apart.
For further information refer to the Product Information.
|Pregnancy category|| |
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.
Luxturna (voretigene neparvovec) was approved for the following therapeutic use:
Luxturna is indicated for the treatment of patients with inherited retinal dystrophy caused by pathological biallelic RPE65 mutations and who have sufficient viable retinal cells as determined by the treating physician.
Pathological mutations of RPE65 should be confirmed by a National Association of Testing Authorities (NATA) or International Laboratory Accreditation Cooperation (ILAC) accredited laboratory.
Luxturna is designed to deliver a normal copy of the gene encoding the human retinal pigment epithelial 65 kDa protein (RPE65) to cells of the retina in persons with reduced or absent levels of biologically active RPE65. The RPE65 is produced in the retinal pigment epithelial (RPE) cells and converts all-trans-retinol to 11-cis-retinol, which subsequently forms the chromophore, 11 cis-retinal, during the visual (retinoid) cycle. The visual cycle is critical in phototransduction, which refers to the biological conversion of a photon of light into an electrical signal in the retina. Mutations in the RPE65 gene lead to reduced or absent levels of RPE65 isomerohydrolase activity, blocking the visual cycle, resulting in impairment of vision and ultimately complete blindness.
The following table summarises the key steps and dates for this application.
|Designation (Orphan)||29 March 2019|
|Submission dossier accepted and first round evaluation commenced||31 July 2019|
|First round evaluation completed||8 January 2020|
|Sponsor provides responses on questions raised in first round evaluation||10 March 2020|
|Second round evaluation completed||15 April 2020|
|Delegate's overall benefit-risk assessment and request for Advisory Committee advice||30 April 2020|
|Sponsor's pre-Advisory Committee response||19 May 2020|
|Advisory Committee meeting||4 and 5 June 2020|
|Registration decision (Outcome)||4 August 2020|
|Completion of administrative activities and registration on ARTG||5 August 2020|
|Number of working days from submission dossier acceptance to registration decision*||208|
*Statutory timeframe for standard applications is 255 working days
- Luxturna (voretigene neparvovec) is to be included in the Black Triangle Scheme. The Product Information (PI) and Consumer Medicines Information (CMI) for Luxturna 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 Luxturna European Union-Risk Management Plan (EU-RMP), version 1.5, dated 4 October 2018 (data lock point 5 May 2017), with Australian specific Annex, version 2.0, dated 26 February 2020), included with submission PM-2019-02585-1-5, to be revised to the satisfaction of 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.
- The treatment centres administering Luxturna should fulfil the following criteria:
- The presence of a specialist ophthalmologist with expertise in care and treatment of patients with inherited retinal dystrophy (IRD).
- The presence of a retinal surgeon experienced in subretinal surgery and capable of administering voretigene neparvovec.
- The presence of a clinical pharmacy capable of handling and preparing adeno-associated virus (AAV) vector based gene therapies.
- Include a clinical geneticist in the multidisciplinary team involved in the care of patients with inherited retinal dystrophy. This condition does not stipulate that the geneticist would need to see all patients at each visit. However, it would be expected that there would be a clinical geneticist involved in the service to assist in the interpretation of tests as required, and oversee appropriate counselling.
- Keep a registry of patients treated with Luxturna (or be involved in the sponsor's registry) which tracks long term efficacy and safety and can identify patients who may need alerting for future safety issues. This registry should include data about vision at baseline, how viable retinal cells were determined, and genotype.
- Batch release testing and compliance with Certified Product Details (CPD)
- All batches of Luxturna imported into/manufactured in Australia must comply with the product details and specifications approved during evaluation and detailed in the Certified Product Details (CPD).
- The sponsor has been granted an exemption by the TGA Laboratories to conduct testing of commercial batches of Luxturna.
This batch release condition will be reviewed and may be modified on the basis of actual batch quality and consistency. This condition remains in place until written notification of any variation is given.
- For all injectable products the PI must be included with the product as a package insert.