The Food and Drug Administration (FDA) has granted Orphan Drug designation to eplontersen for the treatment of patients with transthyretin-mediated amyloidosis.

Transthyretin-mediated amyloidosis (ATTR) is a systemic, progressive and fatal disease caused by the inappropriate formation and aggregations of transthyretin (TTR) amyloid deposits in various tissues and organs. Eplontersen is a ligand-conjugated antisense medicine designed to reduce the production of TTR protein to treat both hereditary and nonhereditary forms of ATTR.

The Company is currently evaluating eplontersen in the phase 3 CARDIO-TTRansform ( Identifier: NCT04136171) and NEURO-TTRansform ( Identifier: NCT04136184) studies for the treatment of hereditary and wild-type amyloid transthyretin cardiomyopathy (ATTR-CM) and hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN), respectively.

“Receiving FDA Orphan Drug status for eplontersen underscores the significant unmet need for novel treatment options for people living with transthyretin-mediated amyloidosis,” said Richard S. Geary, PhD, executive vice president and chief development officer at Ionis. “We look forward to working closely with regulators, clinical investigators, patients and their families to advance this important medicine and make it available to those who may benefit from it.”

The FDA’s Orphan Drug designation is granted to medicines intended to treat or prevent rare diseases or disorders that affect fewer than 200,000 individuals.


Ionis announces eplontersen receives orphan drug designation from US FDA. News release. Ionis Pharmaceuticals, Inc. Accessed January 25, 2022.