EC Approves AstraZeneca/Ionis' Wainzua for Rare Disease ATTRv-PN

AstraZeneca and Ionis Pharmaceuticals have achieved a significant milestone with the approval of their drug, Wainzua (eplontersen), by the European Commission for treating hereditary transthyretin-mediated amyloidosis (ATTRv). This approval targets adults suffering from stage one or two polyneuropathy, known as hATTR-PN or ATTRv-PN. The condition, affecting up to 40,000 individuals globally, is characterized by peripheral nerve damage that results in motor disabilities within approximately five years post-diagnosis.

Hereditary transthyretin (TTR) amyloidosis is a disorder wherein misfolded TTR proteins accumulate in various tissues such as the heart and peripheral nerves, disrupting their normal functionality. This rapid progression can lead to serious cardiovascular, neurological, and renal complications, including heart failure and chronic kidney disease.

Wainzua offers a treatment solution by targeting the liver to reduce TTR protein production. One of its notable features is the convenience of self-administration on a monthly basis through an auto-injector. The decision by the European Commission was influenced by a recommendation from the European Medicines Agency's human medicines committee, bolstered by promising results from the NEURO-TTRansform phase 3 trial. This trial highlighted persistent and consistent benefits for patients using Wainzua, particularly concerning serum TTR concentration and neuropathy impairment, as compared to a placebo group.

Further findings from the trial reflected enhancements in the quality of life of patients, a crucial secondary endpoint, alongside a commendable safety and tolerability profile. Mina Makar, senior vice president for cardiovascular, renal, and metabolism at AstraZeneca, emphasized the significance of this approval as it introduces a new treatment option in the EU. Makar noted that Wainzua provides consistent suppression of TTR production while improving neuropathy impairment and overall quality of life for patients.

In the US, AstraZeneca and Ionis Pharmaceuticals are collaborating to commercialize eplontersen under the brand name Wainua for ATTRv-PN. AstraZeneca retains exclusive rights for both commercializing and developing the drug outside of the US. Brett Monia, the chief executive officer of Ionis, expressed pride in the drug's approval across North America, the UK, and now the EU. He highlighted the collaborative efforts with AstraZeneca to effectively make Wainzua available to patients worldwide.

This approval represents a crucial step forward in the management of hereditary transthyretin-mediated amyloidosis, offering hope to thousands afflicted by this debilitating condition. The drug's ability to address the root cause of TTR protein accumulation and its demonstrated impact on improving patient outcomes underscores the potential of Wainzua as a groundbreaking therapy in the field of rare diseases. As AstraZeneca and Ionis continue their global efforts, the focus remains on delivering effective treatments to enhance the quality of life for individuals grappling with ATTRv-PN.