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AstraZeneca and Ionis’ Wainzua (Eplontersen) Receives the EC’s Approval to Treat Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy

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  • The EC has approved Wainzua to treat ATTRv-PN (stage 1/2 polyneuropathy) in adults pts based on P-III (NEURO-TTRansform) trial assessing eplontersen vs external PBO over 66wks., with follow-up to Wk. 85 & an end-of-trial evaluation. Eligible pts could then enter an ongoing OLE study
  • Trial showed sustained benefits in co-1EPs of serum transthyretin (TTR) levels & neuropathy impairment (mNIS+7) as well as 2EP of QoL (Norfolk QoL-DN) over 66wks.; data was published in The JAMA
  • Eplontersen is being studied in P-III (CARDIO-TTRansform) trial (n=>1,400) for transthyretin-mediated amyloid cardiomyopathy. Both companies are seeking approval in other AstraZeneca-licensed regions

Ref: Businesswire | Image: AstraZeneca & Ionis

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