EU CHMP Endorses AstraZeneca and Ionis' Wainzua for ATTRv-PN

Hereditary transthyretin-mediated amyloidosis with polyneuropathy (ATTRv-PN) is a serious progressive condition that affects peripheral nerves and motor functions. If left untreated, this disease typically results in mortality within ten years. A promising development in the treatment of this condition has emerged from a recommendation by the Committee for Medicinal Products for Human Use (CHMP) of the European Union. The committee has endorsed the approval of Wainzua, a drug developed by AstraZeneca in collaboration with Ionis Pharmaceuticals, for adult patients with stage one or two polyneuropathy caused by ATTRv-PN.

Wainzua's approval would mark a significant milestone as it would become the first self-administered, monthly treatment available in the European Union for this disorder. The medication is administered via an auto-injector, providing patients with a convenient and consistent therapeutic option. The collaboration between AstraZeneca and Ionis Pharmaceuticals, formalized in a global agreement in December 2021, has facilitated the development and potential commercialization of Wainzua. AstraZeneca is responsible for the development and marketing of the drug outside the United States.

Ruud Dobber, the executive vice-president of AstraZeneca's bioPharmaceuticals business unit, highlighted the importance of early diagnosis and innovative therapies for managing hereditary transthyretin-mediated amyloidosis. He emphasized that the progressive nature of ATTRv-PN necessitates timely intervention to help patients better manage the disease, which can otherwise be fatal. Dobber expressed optimism that Wainzua, if approved, would provide a new treatment option that ensures consistent suppression of transthyretin (TTR) levels, ultimately enhancing patients' quality of life.

The positive opinion from the CHMP is grounded in the findings from the NEURO-TTRansform Phase III clinical trial. This trial demonstrated significant and sustained benefits for patients using Wainzua over a period of 66 weeks. The primary focus of the trial was on two main endpoints: the concentration of serum TTR and the degree of neuropathy impairment. Additionally, a crucial secondary endpoint assessed the patients' quality of life. Throughout the trial, Wainzua was noted for its safety and tolerability, reinforcing its potential as a viable treatment option.

Wainzua functions as a once-monthly silencer that targets and suppresses the production of serum TTR at its source in the liver. This mechanism of action is designed to address all forms of ATTR, offering a comprehensive treatment approach. In December 2023, the US Food and Drug Administration approved Wainzua for the treatment of ATTRv-PN under the brand name Wainua. Efforts are underway to secure additional approvals in other global markets.

Furthermore, eplontersen, the active ingredient in Wainzua, has been designated as an orphan drug in both the United States and the European Union. This designation is intended to encourage the development of treatments for rare diseases, providing additional support and incentives to the companies involved.

The potential approval of Wainzua represents a significant advancement in the treatment of hereditary transthyretin-mediated amyloidosis with polyneuropathy. It offers a new, patient-friendly option that could dramatically improve the lives of those affected by this debilitating disease. The ongoing efforts by AstraZeneca and Ionis Pharmaceuticals reflect a commitment to addressing the unmet needs in the management of rare conditions like ATTRv-PN.