VutrisiranAMVUTTRA
Treatment of hereditary transthyretin mediated (hATTR) amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy, defined by Familial Amyloid Neuropathy (FAP) stage.
Decision on record
- Meeting Nov 2024 Recommended Hereditary transthyretin-mediated amyloidosis with polyneuropathy
Access path
- TGA registered · AMVUTTRA
TGA label narrower than the PBS population
- Nov 2024Recommended · restricted
vs patisiran
- PBS listing · Authority Required
From the public summary
7.1 The PBAC recommended the Section 100 (Highly Specialised Drugs Program - Public and Private Hospitals), Authority Required listing of vutrisiran for the treatment of hereditary transthyretin mediated (hATTR) amyloidosis in patients with stage 1 or 2 polyneuropathy.PSD · Nov 2024
7.2 The Committee advised the equi-effective doses were vutrisiran 25 mg given once every 12 weeks and patisiran at a dose of 0.3 mg/kg body weight (maximum 30 mg) 28PSD · Nov 2024
6.42 The submission presented a cost-minimisation of vutrisiran versus patisiran for the treatment of adult patients with hATTR amyloidosis and stage 1 or 2 polyneuropathy, over 18 months (HELIOS-A treatment duration). The submission used a modelled approach, with 3 x 6-monthly cycles (with half-cycle correction), incorporating time- dependent risks of treatment discontinuation and mortality.PSD · Nov 2024
6.43 The key components of the cost-minimisation approach are summarised in Table 9. 17PSD · Nov 2024
6.38 The submission described vutrisiran as comparable in terms of effectiveness compared to patisiran and comparable or superior in terms of safety compared to patisiran. The submission noted that vutrisiran provides a decreased burden for patients, caregivers, and the Australian healthcare system by virtue of its once 3 monthly subcutaneous administration, compared to the patisiran once 3 weekly intravenous infusion.PSD · Nov 2024
6.39 The evaluation considered the therapeutic conclusion presented in the submission was uncertain for the following reasons:PSD · Nov 2024
6.2 The PBAC noted and welcomed the input from individuals (12) and the Leukaemia Foundation. The Committee noted the input from individuals described the burden of living with hATTR amyloidosis and the effectiveness of vutrisiran (as well as patisiran) at improving the symptoms of their condition and slowing the progression of disease.PSD · Nov 2024
The input also outlined the advantages to patients of having a treatment delivered as a subcutaneous injection every 3 months, as opposed to the requirements to attend an infusion centre to receive patisiran once every 3 weeks, in terms of improved ability to work and travel and an increased sense of freedom.PSD · Nov 2024
Cost-effectiveness
Cost-minimisation analysis; no ICER calculated by design.
The submission presented a cost-minimisation of vutrisiran versus patisiran for the treatment of adult patients with hATTR amyloidosis and stage 1 or 2 polyneuropathy, over 18 months (HELIOS-A treatment duration). PSD · 2024
Decision context
PopulationAdult patients aged 18 years or older with hereditary transthyretin amyloidosis confirmed by genetic testing, with stage 1 or stage 2 polyneuropathy (PND score I, II, IIIA, or IIIB), who have not undergone a liver transplant or received previous vutrisiran treatment, and do not exhibit heart failure symptoms (NYHA class III or IV).
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Nov 2024 | Recommended · restricted | patisiran | — | RCT · mNIS+7 composite score |
Consumer voice
Twelve individuals and the Leukaemia Foundation provided input describing the burden of living with hATTR amyloidosis and the effectiveness of vutrisiran at improving symptoms and slowing disease progression. They highlighted the advantages of subcutaneous injection every 3 months compared to frequent infusion centre visits, including improved ability to work, travel, and increased sense of freedo
The input from individuals described the burden of living with hATTR amyloidosis and the effectiveness of vutrisiran (as well as patisiran) at improving the symptoms of their condition and slowing the progression of disease. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to treatment-naïve patients excluding prior vutrisiran, liver transplant recipients, and those with heart failure (NYHA III/IV).