CHMP endorses AstraZeneca/Ionis' Wainzua for rare disease ATTRv-PN

AstraZeneca (AZ) and Ionis' Wainzua (eplontersen) has received a recommendation from the European Medicines Agency’s human medicines committee for its use in adults suffering from hereditary transthyretin-mediated amyloidosis (ATTRv). Specifically, the Committee for Medicinal Products for Human Use (CHMP) has endorsed the drug for the treatment of patients with stage one or two polyneuropathy (PN). This condition impacts multiple nerves in the body, causing pain, progressive weakness, loss of sensation in the limbs, and mobility challenges. It is estimated that up to 40,000 individuals globally are living with ATTRv-PN.

Both hereditary and non-hereditary forms of transthyretin (TTR) amyloidosis occur when the TTR protein accumulates as fibrils in tissues, disrupting their normal functions. As these fibrils build up, tissue damage increases, leading to the worsening of the disease.

Wainzua is designed to reduce the production of the TTR protein directly at its source in the liver. This medication can be self-administered monthly using an auto-injector. The CHMP’s recommendation for Wainzua is based on positive results from the advanced-stage NEURO-TTRansform trial. Patients treated with Wainzua showed consistent and sustained benefits regarding serum TTR concentration and neuropathy impairment when compared to an external placebo group.

Additionally, Wainzua demonstrated improvements in the secondary endpoint of quality of life and maintained a favorable safety and tolerability profile throughout the trial. The European Commission will now review the CHMP’s recommendation to make a final decision on the approval of Wainzua for this indication.

Ruud Dobber, executive vice-president of AstraZeneca's biopharmaceuticals business unit, stated that due to the progressive nature of ATTRv-PN, timely diagnosis and new therapies are crucial for helping patients manage this potentially fatal disease. He emphasized that the CHMP’s recommendation brings Wainzua one step closer to being available for patients in Europe. If approved, it will provide a new treatment option that offers consistent TTR suppression and improves the quality of life for patients.

This positive recommendation follows the recent approval of Wainzua by the Medicines and Healthcare products Regulatory Agency for treating ATTRv-PN. The US Food and Drug Administration also authorized the drug under the brand name Wainua for the same patient group in December of the previous year.

AZ and Ionis are working together to commercialize Wainua for ATTRv-PN in the United States. AstraZeneca will also develop and commercialize the drug in other regions of the world, excluding Latin America.