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Onpattro
Sponsor
Device/Product name
Onpattro
Active Ingredient
Patisiran
Date of decision
Published
Submission type
New chemical entity
ATC codes
N07XX12
Decision
Approved
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 Onpattro 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 B (COR-B) application.
| Description | Date |
|---|---|
| Designation (Orphan) | 12 October 2021 |
| Submission dossier accepted and first round evaluation commenced | 31 January 2022 |
| First round evaluation completed | 17 May 2022 |
| Sponsor provides responses on questions raised in first round evaluation | 14 July 2022 |
| Second round evaluation completed | 21 September 2022 |
| Delegate’s Overall benefit-risk assessment | 15 November 2022 |
| Sponsor’s pre-Advisory Committee response | N/A |
| Advisory Committee meeting | N/A |
| Registration decision (Outcome) | 18 November 2022 |
| Completion of administrative activities and registration on ARTG | 21 November 2022 |
| Number of working days from submission dossier acceptance to registration decision* | 157 |
* The COR-B process has a 175 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
Concentrated injection for infusion
Strength
10 mg/5 mL
Other ingredients
Cholesterol, dibasic sodium phosphate heptahydrate, distearoylphosphatidylcholine, DLin-MC3-DMA, monobasic potassium phosphate, PEG2000-C-DMG, sodium chloride, and water for injections
Containers
Vial
Pack sizes
One
Routes of administration
Intravenous infusion
Dosage
The recommended dose of Onpattro is 300 micrograms per kg body weight administered via intravenous (IV) infusion once every 3 weeks. Dosing is based on actual body weight.
For patients weighing ≥ 100 kg, the maximum recommended dose is 30 mg.
Vitamin A supplementation at approximately 2500 IU vitamin A per day is advised for patients treated with Onpattro (see section 4.4 Special Warnings and Precautions for Use of the Product Information).
For further information refer to the Product Information.
Pregnancy category
D
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.
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
Onpattro (patisiran) was approved for the following therapeutic use:
Onpattro is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy.
What is this medicine and how does it work
Onpattro contains patisiran, a double stranded small interfering ribonucleic acid (siRNA) that targets a genetically conserved sequence in the 3’ untranslated region of a number of variant and wild-type transthyretin messenger ribonucleic acid (TTR mRNA). Patisiran is formulated as lipid nanoparticles to deliver the siRNA to hepatocytes, the primary source of TTR protein in the circulation. Through a process called RNA interference (RNAi), patisiran causes the catalytic degradation of TTR mRNA in the liver, resulting in a reduction of serum TTR protein.
What post-market commitments will the sponsor undertake
- Onpattro (patisiran) is to be included in the Black Triangle Scheme. The PI [Product Information] and CMI [Consumer Medicine Information] for Onpattro 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 Onpattro EU [European Union]-risk management plan (RMP) (version 1.0, dated 25 July 2018, data lock point 14 September 2017), with Australia specific annex (version 0.2, dated 14 July 2022), included with Submission PM-2021-05675-1-1, to be revised to the satisfaction of the TGA, and any subsequent revisions, 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.
- Provide updated data from all ongoing studies as they become available.
- Provide updated post-market data in the form of PSURs/PBRERs [periodic benefit and risk assessment reports] as they become available.
- For all injectable products the Product Information must be included with the product as a package insert.