**AstraZeneca (AZ) and Ionis Pharmaceuticals’ Wainzua (eplontersen) has been approved by the European Commission (EC) to treat a subset of adults with the rare disease hereditary transthyretin-mediated amyloidosis (ATTRv).**
The drug has been authorised for use in patients with stage one or two polyneuropathy, commonly referred to as hATTR-PN or ATTRv-PN. The disease affects up to 40,000 people worldwide and leads to peripheral nerve damage with motor disability within five years of diagnosis.
Transthyretin (TTR) amyloidosis occurs when misfolded TTR protein builds up in tissues, such as the heart peripheral nerves, and interferes with their normal functions. The disease progresses rapidly and can lead to cardiovascular, neurological and renal diseases such as heart failure and chronic kidney disease.
Wainzua, which can be self-administered monthly via an auto-injector, is designed to lower the production of TTR protein at its source in the liver.
The EC’S decision on the drug follows a recent recommendation from the European Medicines Agency’s human medicines committee and was supported by positive results from the phase 3 NEURO-TTRansform trial, in which patients receiving Wainzua demonstrated consistent and sustained benefits on the co-primary endpoints of serum TTR concentration and neuropathy impairment compared to an external placebo group.
Wainzua was also associated with improvements in quality of life, a key secondary endpoint, and demonstrated a favourable safety and tolerability profile throughout the trial.
Mina Makar, senior vice president, cardiovascular, renal and metabolism, AZ, said the approval “marks an important milestone for patients in the EU who can benefit from a new treatment option that provides consistent suppression of TTR production and improves neuropathy impairment and quality of life”.
AZ and Ionis are commercialising eplontersen for ATTRv-PN in the US, where it is approved under the brand name Wainua, while AZ has exclusive commercialisation and development rights in the rest of the world.
Ionis’ chief executive officer, Brett Monia, said: “With approvals in North America, \[the\] UK and now across the EU, we are proud of the continued progress as we and our partner, AZ, rapidly and effectively deliver Wainzua to people around the world.”