Wainua (eplontersen)
Wainua is approved for the treatment of hereditary transthyretin-mediated amyloidosis (ATTRv) in adults with stage 1 or 2 polyneuropathy. Wainua contains the active ingredient eplontersen.
Wainua was approved to treat adults with stage 1 or 2 polyneuropathy caused by hereditary transthyretin amyloidosis (ATTRv).
Polyneuropathy occurs where multiple peripheral nerves outside of the brain and spinal cord are damaged.
Stage 1 polyneuropathy includes patients who can walk without assistance and have mild sensory, motor and autonomic neuropathy in the lower limbs. Stage 2 polyneuropathy includes patients who require assistance with walking and have moderate impairment of the lower limbs, upper limbs, and torso.
Amyloidosis is a group of diseases caused by an abnormal protein called amyloid that builds up in organs. Hereditary transthyretin amyloidosis (ATTRv) is a type of inherited amyloidosis that affects the heart, nervous system and kidneys.
In people with ATTRv, the protein transthyretin (TTR) is defective and breaks easily. This causes it to bunch together and form amyloid deposits. Amyloid deposits can build up in or around nerves and other parts of the body, stopping them from working properly.
Wainua works by reducing how much TTR protein the liver makes. This means there is less TTR protein in the blood to build up as amyloid deposits and this can help to reduce disease symptoms.
The effectiveness and safety of Wainua were assessed in one main clinical study and several supporting studies.
The main study compared the effectiveness and safety of Wainua to a placebo (treatment with no active medicine) in adult patients with stage 1 or stage 2 polyneuropathy.
Effectiveness of Wainua was determined by the following:
- percentage change in blood TTR concentration
- changes in nerve damage measured using the modified Neuropathy Impairment Score plus 7 (mNIS+7) composite score
- changes in quality of life measured using the Norfolk Quality of Life - Diabetic Neuropathy (Norfolk QoL-DN) total score.
Key findings from clinical studies
- Blood TTR concentration reduced by 81.7% in patients treated with Wainua compared to 11.2% in patients given the placebo. The reduction in blood TTR concentration was maintained throughout treatment and was consistent across all patient demographics.
- Wainua showed an increased benefit when compared to the placebo. Improvement in mNIS+7 score was observed in 48.2% of patients in the Wainua group compared to 16.9% in the placebo group.
- An improvement in patient-reported quality of life was shown. In the Wainua group, 58.9% of patients showed an improvement in Norfolk QoL DN total score compared to 20.3% in the placebo group.
- The most frequent side effects of Wainua were decreased vitamin A, vomiting, and redness (erythema), itching (pruritus) and pain where the injection was given.
The TGA decided that the application provided sufficient evidence supporting the safety and effectiveness of Wainua and the medicine can be registered in Australia.
More detailed information on why the TGA approved Wainua will be published in the upcoming Australian Public Assessment Report (AusPAR).
For comprehensive information on potential side effects and risks, refer to the Consumer Medicine Information (CMI) leaflet or Product Information (PI) document.
This decision summary will not be updated to reflect any subsequent changes and may therefore not contain the most current information about the medicine. For the latest information, refer to the medicine’s CMI or PI.
The CMI leaflet offers guidance for consumers to support safe and effective use of the medicine. The CMI includes information on dose, how to use the medicine properly, potential side effects, safety precautions, storage instructions and more.
Australian CMIs can be accessed through the searchable TGA eBusiness Services or ARTG databases.
The PI document provides essential prescribing information for health professionals, including details on dosage recommendations, pregnancy category, contraindications, precautions and potential side effects.
Australian PIs can be accessed through the searchable TGA eBusiness Services or ARTG databases.
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Before starting treatment with Wainua, a patient's vitamin A levels will be checked.
Patients receiving Wainua should take oral vitamin A supplements during treatment as this medicine lowers the amount of vitamin A in the blood.
If patients develop problems with vision or other eye problems while taking Wainua, referral to an eye specialist is recommended.