On October 21, AstraZeneca and Ionis announced that Wainzua (Eplontersen) had received a positive recommendation for approval from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP).For the treatment of patients with Stage 1 or Stage 2 polyneuropathy(Usually referred to as hATTR-PN or ATTRv-PN)Hereditary Transthyretin-Mediated Amyloidosis in Adult Patients。The press release pointed out,If approved by the European Commission,Eplontersen Will become the only EU-approved drug for treating ATTRv-PN, patients can self-administer the injection monthly via an auto-injector.
Screenshot source: AstraZeneca official website
Transthyretin-Mediated Amyloidosis (ATTR) is caused by the accumulation of misfolded TTR protein, derived from the liver, in tissues such as the heart and peripheral nerves, leading to organ damage and failure. ATTR subsequently causes complications that result in cardiovascular, neurological, and renal diseases, such as heart failure and chronic kidney disease. ATTR has two forms: hereditary (ATTRv) and non-hereditary (wild-type).ATTRv-PN is a debilitating disease that causes peripheral nerve damage and motor impairment within five years of diagnosis and is usually fatal within a decade if left untreated.。EplontersenIt is an antisense oligonucleotide (ASO) therapy developed by Ionis Pharmaceuticals, Inc. that inhibits the production of TTR and is administered once a month.It is an RNA-targeted drug designed to reduce the production of TTR protein in the liver, thus having the potential to treat all types of ATT.R。In 2021, Ionis Pharmaceuticals and AstraZeneca entered into a collaboration to jointly develop and commercialize Eplontersen in the United States, while granting AstraZeneca the rights to develop and commercialize the drug in regions outside of Latin America.The total amount of this cooperation exceeds 3.5 billion US dollars.. In July 2023, the two parties expanded their cooperation again, and AstraZeneca obtained the exclusive rights to commercialize Eplontersen in Latin America.The total amount of this cooperation also exceeds 3.6 billion US dollars.。Previously,EplontersenEplontersen was approved by the U.S. FDA in December of last year for the treatment of ATTRv-PN. In September this year, Eplontersen also filed for marketing approval in China. Based on the research progress of this drug in China, it is speculated that its indication filed in China may be: for adult patients with transthyretin amyloid cardiomyopathy (ATTR-CM).The CHMP's approval opinion this time is based on the positive NEURO-TTRansform Phase III trial. The trial data showed that, compared with placebo, within 66 weeks, those receivingEplontersenPatients treated inSerum Transthyretin (TTR) ConcentrationAndNeuropathic InjuryTheCo-primary EndpointAndKey Secondary Endpoint of Quality of Life (QoL)Aspect, showing consistent and sustained benefits. Moreover, throughout the NEURO-TTRansform trial,EplontersenContinued to demonstrate good safety and tolerability.
Screenshot source: Insight database
Cover Source:Corporate Logo
Disclaimer:This article is for information sharing only,不代表 Insight 立场和观点，也不作治疗方案推荐和介绍。如有需求，请咨询和联系正规医疗机构。
Editor: Xinyao
PR Article Coordination: WeChat insightxb
SubmissionWeChat: insightxb; Email: insight@dxy.cn
Diversified functions, traceable data……Insight Database Web Version Awaits Your Experience
Click to read the original text,ImmediatelyUnlock！
