AstraZeneca and Ionis Pharmaceuticals’ polyneuropathy drug has won approval in the European Union (EU), where it will be marketed as Wainzua (eplontersen).
The drug has been approved to treat hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy (ATTRv-PN). This marks the second major regulatory clearance for the therapy, following its approval in the US in December 2023, where it is sold as Wainua.
The companies have positioned Wainzua as a differentiated treatment for ATTRv-PN, as it is the first therapy for the condition that can be self-administered using a once-monthly pen injector. Other available therapies typically require administration by a healthcare professional.
Wainua is a ligand-conjugated antisense oligonucleotide (ASO) that works by reducing the production of misfolded transthyretin (TTR) protein. In hereditary transthyretin-mediated amyloidosis, the accumulation of these misfolded proteins leads to amyloid fibril deposits that cause progressive tissue damage, resulting in neurological impairment. This causes symptoms like pain, numbness, and weakness.
The drug enters a competitive market currently led by Alnylam Pharmaceuticals, which markets two synthetic small interfering RNA (siRNA) therapies for ATTRv-PN – Onpattro (patisiran) and Amvuttra (vutrisiran). Amvuttra, which secured EU approval in 2022, is administered quarterly via a prefilled syringe, rather than Wainua’s monthly self-injection.
Ionis already has experience in the ASO space, with its first-generation therapy Tegsedi (inotersen) also approved for ATTRv-PN. However, Tegsedi requires weekly subcutaneous injections and comes with additional safety monitoring requirements due to potential thrombocytopaenia and glomerulonephritis risks. Ionis removed Tegsedi from the US market in 2023, but it continues to be marketed as a treatment option for ATTRvPN in the EU. This could make Wainzua a more favourable option among patients and healthcare providers.
The European Commission’s decision to approve Wainzua was supported by data from the Phase III NEURO-TTRansform study (NCT04136184). At 66 weeks, the study showed that patients receiving Wainzua experienced a “consistent and sustained” benefit in neurologic impairment – as determined by a neuropathy impairment score +7 (mNIS+7) – and serum transthyretin (TTR) concentration.
The ATTRv-PN market has seen significant revenue growth in recent years. Alnylam’s Amvuttra generated $970m in 2024, as per the company financials. Wainzua generated sales of $85m in 2024, but is forecast to pull in up to $2.2bn in sales by 2030, according to GlobalData’s Pharma Intelligence Center. Amvuttra is set to make double this in the same year with $4.4bn in projected sales.
GlobalData is the parent company of Pharmaceutical Technology.
