hereditary transthyretin amyloidosis
Rare disease · 2023–2024
Every decision
| Medicine | Meeting | Outcome | Consumer input |
|---|---|---|---|
| vutrisiran Treatment of hereditary transthyretin mediated (hATTR) amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy, defined by Familial Amyloid… | Nov 2024 | Recommended with restriction | 12 individuals Leukaemia Foundation |
| patisiran Hereditary transthyretin-mediated amyloidosis (hATTR) with stage 1 or stage 2 polyneuropathy in adult patients. | Dec 2023 | Not recommended | 57 individuals Australian Amyloidosis Network |
Who spoke
Australian Amyloidosis Network · Leukaemia Foundation
What the PBS pays
$2M in government benefit over 2024–25, across 15 services.
This is spend on the medicines considered for this condition, not spend on the disease. A medicine used for several conditions is counted in full under each, so figures across conditions add to more than national PBS expenditure.
| Medicine | Government benefit |
|---|---|
| vutrisiran | $1.7M |
Appraised elsewhere
NICE, in England and Wales, has appraised 1 technology for this condition, recommending 1 . A different committee, a different population and a different price: this is what has been looked at there, not a verdict on what happened here.
| Technology | Appraisal | Outcome | Date |
|---|---|---|---|
| eplontersenEplontersen for treating hereditary transthyretin-related amyloidosis | TA1020 | recommended restricted | 2024-11 |
Conditions are classified by a language model from the indication text of each submission, so an individual label here can be wrong. Consumer input is read from the document's own consumer comments section: 2 of 2 decisions since 2014 carried some. Silence means no comment reached the committee through that facility, not that nobody was affected. More on how this was built.