Although we won't be issuing a formal press release, we wanted to
let you know NICE has today (Tuesday, 29 October) published
guidance recommending a new drug which can be taken at home for
people with a rare inherited condition.
Eplontersen, also known as Wainzua and made by AstraZeneca, is
recommended as an option for treating hereditary
transthyretin-related amyloidosis in adults with stage 1 or stage
2 polyneuropathy.
Transthyretin-related amyloidosis occurs when small fibres of TTR
protein clump together to make deposits called ‘amyloid.' Amyloid
can build up around or within the nerves, heart, and other
organs, stopping them from working properly.
Eplontersen is specifically indicated for use when the disease is
causing polyneuropathy, which is damage to multiple nerves
outside of the brain and central nervous system, resulting in
pain, discomfort, progressive weakness, and loss of sensation in
the legs and arms, and mobility difficulties.
The treatment, which is given as an injection under the skin
using a pre-filled pen, works by lowering the amount of TTR
protein made by the liver. As a result, there is less TTR protein
in the blood to form amyloid deposits, helping to reduce the
effects of the disease.
It is estimated that around 12 people in England could benefit
from this new treatment option in its first year. In the UK there
are around 160 people who are diagnosed with the disease and
receive treatment.
Eplontersen was found to be similarly effective compared to
vutrisiran, another treatment for the same condition. Clinical
evidence showed that in people with stage 1 or 2 polyneuropathy
eplontersen appears to be more effective than placebo at reducing
serum transthyretin levels and increases how long they have
before their polyneuropathy gets worse.
NICE has published final draft guidance on eplontersen within two
weeks of the treatment being granted its marketing authorisation
by the Medicines and Healthcare products Regulatory Agency
(MHRA), demonstrating NICE's commitment to getting the best care
to patients fast.
As eplontersen has been recommended through the cost-comparison process, NHS
England and integrated care boards have agreed to provide funding
to implement the guidance 30 days after publication of final
guidance.
Read the full final draft guidance for eplontersen for treating
hereditary transthyretin-related amyloidosis.
