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

U.S.-listed shares of AstraZeneca /zigman2/quotes/200304487/composite AZN -0.34% 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. /zigman2/quotes/201272020/composite IONS -2.77% 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 /zigman2/quotes/210599714/realtime SPX +1.45% has tumbled 7.7% year-to-date.

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AstraZeneca PLC ADR

US : U.S.: Nasdaq

$ 72.59

-0.25 -0.34%

Volume: 8.04M

June 2, 2023 4:00p

P/E Ratio

48.10

Dividend Yield

1.96%

Market Cap

$227.28 billion

Rev. per Employee

$525,018

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Ionis Pharmaceuticals Inc.

US : U.S.: Nasdaq

$ 41.75

-1.19 -2.77%

Volume: 959,017

June 2, 2023 4:00p

P/E Ratio

N/A

Dividend Yield

N/A

Market Cap

$5.97 billion

Rev. per Employee

$723,721

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S&P 500 Index

US : S&P US

4,282.37

+61.35 +1.45%

Volume: 0.00

June 2, 2023 5:21p