The US FDA has granted eplontersen, an antisense oligonucleotide (ASO) targeting TTR production, Fast Track designation for the treatment of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) in adults.

ATTR-CM is an underdiagnosed, progressive and potentially fatal disorder caused by the accumulation of misfolded TTR protein in the cardiac muscle. ATTR-CM typically leads to progressive heart failure and death within three to five years after disease onset.

Recently, eplontersen has received approval for the treatment of polyneuropathy in patients with hereditary ATTR. Eplontersen is currently being evaluated for heredity or wild-type ATTR-CM in the double-blind, randomized, placebo-controlled, phase 3 CARDIO-TTRansform study, which finished enrollment of more than 1400 patients. First results of CARDIO-TTransform are expected in 2025.

The FDA Fast Track designation facilitates the development of potential new therapies to treat serious conditions and fulfil an unmet medical need.

Source: Press release Ionis Pharmaceuticals, Feb 8, 2024