Ionis announces eplontersen receives orphan drug designation from U.S. FDA

CARLSBAD, Calif.: CARLSBAD, Calif., Jan. 24, 2022 /PRNewswire/ — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS), the leader in antisense therapeutics, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to eplontersen, an investigational antisense medicine for the treatment of people living with transthyretin-mediated amyloidosis, a systemic, progressive and fatal condition. Orphan drug designation is granted by the FDA to drugs and biologics intended for treatment, prevention or diagnosis of a rare disease or condition that affects fewer than 200,000 people in the U.S. at the time of designation.
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