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Ionis Pharma Says FDA Grants Orphan Drug Designation To Eplontersen - Quick Facts

Ionis Pharmaceuticals, Inc. (IONS) announced Monday 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.

Under the FDA's Orphan Drug Act, orphan drug status provides incentives, including tax credits, grants and waiver of certain administrative fees for clinical trials, and seven years of market exclusivity following drug approval.

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