Amvuttra (vutrisiran)
Key trials in humans that supported the proposed indication included a Phase 1 single-dose study in healthy volunteers and a pivotal Phase 3 study in hATTR amyloidosis patients with polyneuropathy.
The active ingredient with its proposed indication was given orphan drug designation and was evaluated under the Comparable Overseas Regulator B (COR-B) report-based process.
| Description | Date |
|---|---|
| Designation (Orphan) | 28 August 2023 |
| Submission dossier accepted and first round evaluation commenced | 31 October 2023 |
| First round evaluation completed | 29 February 2024 |
| Sponsor provides responses on questions raised in first round evaluation | 28 March 2024 |
| Second round evaluation completed | 22 April 2024 |
| Delegate’s Overall benefit-risk assessment | 15 May 2024 |
| Registration decision (Outcome) | 18 June 2024 |
| Administrative activities and registration in the ARTG completed | 21 June 2024 |
| Number of working days from submission dossier acceptance to registration decision* | 161 |
*The COR-B process has a 175 working day evaluation and decision timeframe.
for Amvuttra must include the black triangle symbol and mandatory accompanying text
for five years, which starts from the date of first supply of the product.
- Monobasic sodium phosphate dihydrate
- Dibasic sodium phosphate dihydrate
- Sodium chloride
- Water for injections
- Sodium hydroxide (for pH adjustment)
- Phosphoric acid (for pH adjustment)
The recommended dose of Amvuttra is 25 mg administered via subcutaneous injection once every 3 months.
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.
Amvuttra was approved for the treatment of hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy.
TTR transports vitamin A (retinol) and a hormone called thyroxine throughout the body. To perform this transport function, TTR units must form a tetramer (a protein complex made up of four TTR subunits). Patients with hATTR possess TTR gene variants that prevent tetramer formation and form fibril clumps instead which form amyloid. These amyloid deposits cause the symptoms of hATTR by accumulating in certain tissues. Amvuttra alleviates these symptoms by reducing TTR synthesis which leads to reduced levels of amyloid deposits.
The Amvuttra Risk Management Plan (RMP) as agreed with the TGA will be implemented in Australia. An obligatory component of RMPs is routine pharmacovigilance, which includes the submission of periodic safety update reports.
More information
The latest Product Information (PI) and Consumer Medicine 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.
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