The Medicines and Healthcare products Regulatory Agency (MHRA)
has approved eplontersen (Wainzua) to treat adults with
polyneuropathy associated with hereditary transthyretin
amyloidosis (ATTRv), which is a rare progressive condition that
runs in families caused by a protein called transthyretin (TTR).
In people with this disease, 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.
Eplontersen works mainly 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. That can help reduce the effects
of the disease.
The recommended dose of eplontersen is one 45mg dose every month,
administered as an injection under the skin using a pre-filled
pen. Treatment with eplontersen lowers the amount of vitamin A in
the blood and patients will need to take vitamin A supplements
during treatment.
Beach, MHRA Interim Executive
Director of Healthcare Quality and Access, said:
Enabling safe access to high quality, safe and effective
medicines is a key priority for us.
We're assured that the appropriate regulatory standards of
safety, quality and effectiveness for the approval of this new
formulation have been met.
As with all products, we will keep its safety under close
review.
The MHRA's approval of the medicine is supported by evidence from
a NEURO-TTRansform study. In this trial 164 adult patients with
hereditary transthyretin-mediated amyloidosis received a
subcutaneous injection of eplontersen 45 mg every 4 weeks
compared to an historical placebo arm from the older NEURO-TTR
study completed in 2017 as well as patients who received another
medicine called inotersen 284 mg weekly.
The study looked at levels of TTR in the blood of patients to
measure the levels of this protein that contributes to the
disease. It also used questionnaires to rate the changes that
patients reported in the symptoms of their nerve damage. This
study tracked these changes from the start of the trial
(baseline) to 35 and 66 weeks after the patients had received
eplontersen.
Patients in the trial receiving eplontersen experienced greater
reductions in TTR levels and less worsening of the disease from
baseline compared to the placebo group.
A full list of all side effects reported with this medicine is
available in the patient information leaflet or from the product
information published on the MHRA website.
If a patient experiences any side effects, they should talk to
their doctor, pharmacist, or nurse. This includes any possible
side effects not listed in the product information leaflets.
Anyone who suspects they are having a side effect from this
medicine is encouraged to talk to their doctor, pharmacist or
nurse and report it directly to the MHRA's Yellow Card scheme.
ENDS
Notes to editors
- The new marketing authorisation was granted for eplontersen
(Wainzua) on 14 October to AstraZeneca via National Procedure.
