EU Approves AstraZeneca and Ionis for ATTR Polyneuropathy

AstraZeneca and Ionis Pharmaceuticals have achieved a significant milestone with the European Union's approval of their innovative polyneuropathy medication, Wainzua (eplontersen). This new treatment is designed for adult patients with hereditary transthyretin-mediated amyloidosis (ATTRv-PN), specifically those in stage 1 or stage 2 of the disease. The EU approval follows the earlier clearance of the drug in the United States in December 2023, where it is marketed under the name Wainua.

Wainzua stands out in the field of ATTRv-PN therapies primarily due to its user-friendly administration method. It is the first treatment for this condition that can be self-administered via a once-monthly pen injector, offering a significant advantage over other treatments that require administration by healthcare professionals. This ease of use could enhance patient compliance and improve overall treatment outcomes.

The mechanism of action for Wainzua involves a ligand-conjugated antisense oligonucleotide (ASO), which functions by reducing the production of misfolded transthyretin (TTR) protein. In patients with hereditary transthyretin-mediated amyloidosis, the abnormal accumulation of these proteins results in amyloid fibril deposits, leading to progressive tissue damage and neurological impairment. Symptoms often include pain, numbness, and muscle weakness, significantly impacting the quality of life.

Wainzua enters a competitive market where Alnylam Pharmaceuticals currently leads with its two synthetic small interfering RNA (siRNA) therapies for ATTRv-PN: Onpattro (patisiran) and Amvuttra (vutrisiran). Amvuttra, which received EU approval in 2022, is administered quarterly using a prefilled syringe, contrasting with Wainua's monthly self-injection approach.

Ionis Pharmaceuticals, a key player in the ASO domain, has previously introduced Tegsedi (inotersen) for ATTRv-PN. However, Tegsedi comes with the inconvenience of weekly subcutaneous injections and necessitates additional safety monitoring due to risks of thrombocytopaenia and glomerulonephritis. Ionis decided to remove Tegsedi from the US market in 2023 while continuing to offer it in the EU. This decision positions Wainzua as a potentially more appealing option for both patients and healthcare providers, given its improved safety profile and convenience.

The European Commission based its approval of Wainzua on the compelling results from the Phase III NEURO-TTRansform study (NCT04136184). The 66-week trial demonstrated that patients receiving Wainzua experienced a "consistent and sustained" improvement in neurological impairment, assessed by a neuropathy impairment score +7 (mNIS+7), alongside maintaining stable serum transthyretin (TTR) levels.

The market for ATTRv-PN treatments has witnessed robust growth recently. Alnylam's Amvuttra achieved impressive sales of $970 million in 2024, according to the company's financial reports. In contrast, Wainzua's sales were $85 million in the same year. Nonetheless, projections from GlobalData’s Pharma Intelligence Center anticipate that Wainzua could generate up to $2.2 billion in sales by 2030, while Amvuttra is expected to reach $4.4 billion.

With its recent approval, Wainzua is poised to become a significant player in the ATTRv-PN market, offering a promising treatment alternative for patients and potentially capturing a substantial market share in the years to come.