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Investor Alert

Jan. 24, 2022, 7:51 a.m. EST

AstraZeneca says drug for rare, fatal condition gets orphan-drug designation

U.S.-listed shares of AstraZeneca (NAS:AZN) were down 1.% in premarket trading on Monday after the company said it received an orphan-drug designation for its experimental treatment for transthyretin-mediated amyloidosis, a rare condition that causes heart failure and death within years of diagnosis. The treatment, eplontersen, comes from a development and commercialization deal that AstraZeneca inked with Ionis Pharmaceuticals Inc. (NAS:IONS) in December. AstraZeneca said it aims to file the drug for approval with the Food and Drug Administration by the end of 2022. Ionis shares were down 0.2% in premarket trading. The FDA can give orphan-drug designations to therapies designed to treat diseases or disorders that affect fewer than 20,000 people in the U.S. AstraZeneca's stock is up 1.4% for the year, while Ionis shares have declined 1.8%. The S&P 500 (S&P:SPX) has tumbled 7.7% year-to-date.

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