How Wainua Treats hATTR Amyloidosis with Polyneuropathy
Wainua (eplontersen) is an antisense oligonucleotide injected subcutaneously once every 4 weeks. It targets the transthyretin (TTR) gene's mRNA in the liver, binding to it and triggering RNAse H-mediated degradation. This reduces hepatic production of TTR protein by about 80-90% from baseline, preventing formation of toxic TTR amyloid fibrils that deposit in peripheral nerves and cause polyneuropathy symptoms like pain, numbness, weakness, and autonomic dysfunction.[1][2]
By stabilizing TTR tetramers and halting new amyloid buildup, Wainua slows neuropathy progression, improves neuropathy impairment scores (e.g., mNIS+7 scale), and enhances quality of life compared to placebo, as shown in the NEURO-TTRansform phase 3 trial (18-month data).[1][3]
How Quickly Does It Start Working?
Neuropathy stabilization occurs within 3-6 months, with peak TTR reduction by week 13. In trials, 47% of patients on Wainua had no progression vs. 8% on placebo at 15 months.[1][3]
What Are Common Side Effects?
Most are mild: injection-site reactions (30%), arthralgia, diarrhea. Vitamin A supplementation is required due to TTR's role in its transport, with monitoring for deficiency. No increased mortality or severe liver toxicity reported.[1][2]
How Does It Compare to Vutrisiran (Amvuttra) or Patisiran (Onpattro)?
All three lower TTR similarly (80-90%), but Wainua's self-administered subcutaneous dosing contrasts with IV-infused patisiran (every 3 weeks) or subcutaneous vutrisiran (every 3 months). NEURO-TTRansform showed Wainua superior to placebo on mNIS+7 (-0.25 point change vs. +7.68); head-to-head data pending.[1][4]
| Drug | Dosing | TTR Reduction | Trial Polyneuropathy Benefit |
|------|--------|---------------|-----------------------------|
| Wainua | SC Q4W | ~85% | Better than placebo [3] |
| Amvuttra | SC Q3M | ~85% | Better than placebo [4] |
| Onpattro | IV Q3W | ~80% | Better than placebo [4] |
Who Makes Wainua and What's the Cost?
AstraZeneca and Ionis Pharmaceuticals co-developed it; FDA/EMA approved 2024 for polyneuropathy of hereditary ATTR amyloidosis in adults.[1][5] U.S. list price ~$463,000/year; patient assistance programs available.[6]
When Do Patents Expire?
Key U.S. patents on eplontersen composition/methods extend to 2038-2041, per DrugPatentWatch; no generics imminent.[7]
Sources
[1]: Wainua Prescribing Information (AstraZeneca)
[2]: NEJM: NEURO-TTRansform Trial
[3]: AstraZeneca Press Release (2024)
[4]: FDA Labels: Amvuttra/Onpattro
[5]: EMA Approval Summary
[6]: GoodRx Pricing Data
[7]: DrugPatentWatch: Eplontersen