Eplontersen and vutrisiran for hereditary transthyretin amyloidosis with polyneuropathy
Cardiovascular
Neurology
1 July 2026
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Published on 06 Feb 2026
Last Updated on 01 Jul 2026
Guidance Recommendations
The Ministry of Health’s Drug Advisory Committee has recommended:
Vutrisiran 25 mg/0.5 mL injection for stage 1 or 2 hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN).
Funding status
Vutrisiran 25 mg/0.5 mL injection is recommended for inclusion on the Medication Assistance Fund (MAF) for the abovementioned indication from 1 September 2026.
Patient must be undergoing treatment with vutrisiran as a monotherapy (i.e. not in combination with any other disease modifying medicines for amyloidosis disorders), and treatment should be used in line with the additional clinical criteria listed in the Annex.
MAF assistance does not apply to any formulations or strengths of eplontersen for treating hATTR-PN.
Plain English Summary
Hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) is an inherited condition (passed down through families) caused by changes (mutations) in the transthyretin (TTR) gene. These genetic changes lead to abnormal transthyretin proteins that build up in the body, forming deposits that damage organs and nerves. The condition usually develops in adulthood and most commonly affects the nerves in the hands and legs that control feeling and movement. It can also affect nerves that control body functions such as digestion and blood pressure. In some people, the heart may also be affected.
Nerve damage caused by hATTR-PN worsens over time. Doctors describe the condition in stages ranging from stage 0 (no symptoms) to stage 3 (severe impairment). In stage 1, people can walk without help but may have mild nerve problems, mainly in the lower limbs. In stage 2, people need help to walk and have more widespread nerve problems affecting the lower limbs, upper limbs, and trunk.
Eplontersen and vutrisiran are medicines known as transthyretin silencers. They work by reducing the production of the abnormal transthyretin protein that causes nerve damage in hATTR-PN. Both medicines are given as injections under the skin (subcutaneously) and can be self-administered at home.
Vutrisiran was recommended for inclusion in the Medication Assistance Fund (MAF) for government subsidy because its benefit justifies its cost. The treatment cost will be subsidised by 40% to 75% for eligible patients. Eplontersen was not recommended for subsidy as its benefits do not justify its cost compared with vutrisiran.
