Avidity Biosciences Gets Orphan-Drug Status for Muscular Dystrophy Treatment
15 Agosto 2023 - 10:41AM
Dow Jones News
By Dean Seal
Avidity Biosciences has gotten orphan drug designation from
regulators for its investigational therapy for the treatment of
Duchenne muscular dystrophy.
The biopharmaceutical company said the U.S. Food and Drug
Administration granted the designation to AOC 1044, which is being
developed to treat Duchenne muscular dystrophy in people with
mutations amenable to exon 44 skipping.
Orphan-drug designation is a special status given to drugs that
show promise for potentially treating rare, or orphan, diseases
that have fewer than 200,000 cases a year in the U.S.
Shares ticked up 1.8% to $8.93 in premarket trading.
Write to Dean Seal at dean.seal@wsj.com
(END) Dow Jones Newswires
August 15, 2023 09:26 ET (13:26 GMT)
Copyright (c) 2023 Dow Jones & Company, Inc.
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