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Highlights:
- Wainzua recommended for EU approval as the first self-administered treatment for hereditary transthyretin-mediated amyloidosis.
- Phase III NEURO-TTRansform trial shows significant improvements in neuropathy impairment and quality of life.
- Experts emphasize the urgent need for new therapies to combat this debilitating and potentially fatal disease.
AstraZeneca and Ionis’ Wainzua (eplontersen) has received a positive recommendation for approval from the Committee for Medicinal Products for Human Use (CHMP) in the European Union (EU). This groundbreaking treatment targets hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients experiencing stage 1 or stage 2 polyneuropathy (hATTR-PN). If approved by the European Commission, Wainzua will become the first approved medication in the EU that allows for self-administration through a monthly auto-injector.
The CHMP’s recommendation is rooted in the results of the NEURO-TTRansform Phase III trial, which demonstrated that Wainzua provided consistent and sustained benefits over a 66-week period. Patients treated with Wainzua showed significant improvements in both the co-primary endpoints of serum transthyretin (TTR) concentration and neuropathy impairment, as measured by the modified Neuropathy Impairment Score +7 (mNIS+7). Additionally, key secondary outcomes reflected enhancements in quality of life, as assessed by the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN), compared to an external placebo group. Importantly, the treatment maintained a favorable safety and tolerability profile throughout the study.
Hereditary transthyretin-mediated amyloidosis is a severe and often fatal condition that can lead to substantial peripheral nerve damage within five years of diagnosis. Without effective treatment, the prognosis can be dire, typically resulting in death within a decade. Experts in the field are eager for new therapeutic options, as Dr. Laura Obici, Head of the Rare Diseases Unit in Italy, highlights: “This debilitating disease is ultimately fatal if left untreated. Additional treatment options designed to reduce the production of TTR protein at its source could offer patients more time and improve their quality of life.”
Ruud Dobber, Executive Vice-President of AstraZeneca’s BioPharmaceuticals Business Unit, also emphasized the urgency of timely diagnosis and the need for innovative therapies. He noted, “Today’s recommendation brings Wainzua one step closer for patients in Europe, offering a new treatment option that can provide consistent TTR suppression and improve quality of life.”
Wainzua functions as a once-monthly silencer, specifically designed to suppress TTR production at its source in the liver. The treatment targets all types of transthyretin-mediated amyloidosis, representing a significant advancement in therapeutic options for patients. In December 2023, Wainzua received approval under the brand name Wainua in the United States, and it is now progressing through the regulatory process in various countries worldwide.
Moreover, eplontersen is currently under evaluation in the CARDIO-TTRansform Phase III trial, aimed at treating transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), which is the largest study of its kind to date, involving over 1,400 participants.
As the EU continues to assess this promising treatment, stakeholders eagerly await the final decision from the European Commission. The potential approval of Wainzua represents not just a therapeutic milestone but also a beacon of hope for patients grappling with hATTR-PN, underscoring the vital role that innovative therapies play in improving patient outcomes and quality of life.
