Ioniis: Eplontersen receives orphan drug designation

 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.

Eplontersen is a LIgand-Conjugated Antisense (LICA) investigational medicine currently in Phase 3 clinical trials for amyloid transthyretin cardiomyopathy (ATTR-CM) and amyloid transthyretin polyneuropathy (ATTR-PN). It is designed to reduce the production of transthyretin (TTR protein) to treat both hereditary and non-hereditary forms of TTR amyloidosis (ATTR). In December 2021, Ionis announced a strategic collaboration with AstraZeneca to develop and commercialize eplontersen.

https://finance.yahoo.com/news/ionis-announces-eplontersen-receives-orphan-120500840.html