Wainzua recommended for approval in the EU for the treatment of adult patients with polyneuropathy associated with hereditary transthyretin-mediated amyloidosis
Posted on October 28, 2024
AstraZeneca and Ionis’ Wainzua have recently been recommended for approval by the Committee for Medicinal Products for Human Use in the European Union for the treatment of hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy, commonly referred to as hATTR-PN or ATTRv-PN. If approved by the European Commission, Wainzua will be the only approved medicine in the EU for the treatment of ATTRv-PN that can be self-administered monthly via an auto-injector.
The CHMP based its opinion on the positive NEURO-TTRansform Phase III trial which showed that through 66 weeks, patients treated with Wainzua demonstrated consistent and sustained benefit on the co-primary endpoints of serum transthyretin concentration and neuropathy impairment measured by modified Neuropathy Impairment Score +7 , and key secondary endpoint of quality of life on the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy versus external placebo.
Dr Laura Obici, Head of Rare Diseases Unit, Consultant at the Amyloidosis Research and Treatment Centre Istituto Di Ricovero e Cura a Carattere Scientifico Fondazione Policlinico San Matteo, Pavia, Italy, said: “This debilitating disease is ultimately fatal if left untreated and can have a significant impact on many aspects of patients’ and caregivers’ day-to-day lives. Having additional amyloidosis treatment options designed to reduce the production of TTR protein at its source would potentially give patients more time and ability to do what matters most to them and offer the hope of living longer with a higher quality of life.”
Ruud Dobber, Executive Vice-President, BioPharmaceuticals Business Unit, AstraZeneca, said: “Due to the progressive nature of polyneuropathy of hereditary transthyretin-mediated amyloidosis, it is critical to have timely diagnosis and new therapies to help people have greater control over this potentially fatal disease. Today’s recommendation brings Wainzua one step closer for patients in Europe, and if approved, will offer a new treatment option that can provide consistent TTR suppression and results in improved quality of life.”
Related Topics and Keywords
CHMP, polyneuropathy, transthyretin-mediated amyloidosis, Wainzua (eplontersen)
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