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FDA Accepts Ionis NDA for Eplontersen in Rare Hereditary Disease

FDA Accepts Ionis NDA for Eplontersen in Rare Hereditary Disease

The FDA has accepted Ionis Pharmaceuticals’ new drug application (NDA) for eplontersen to treat patients with hereditary transthyretin-mediated amyloid polyneuropathy, a rare inherited genetic condition.

The condition causes a protein called transthyretin (TTR) to misfold into amyloid deposits, causing nerve damage throughout the body and progressive loss of motor function. TTR also accumulates in major organs, causing them to lose function and leading to death within five to 15 years after disease onset. 

Eplontersen is an investigational medicine designed to reduce transthyretin protein production in both hereditary and non-hereditary forms of transthyretin amyloidosis, the company said.

The NDA is supported with results from a phase 3 study that showed significant mean reduction in patients’ serum transthyretin concentration compared to baseline.

About Ionis Pharmaceuticals, Inc.

The FDA’s target action date on the NDA is Dec. 22.

Ionis Pharmaceuticals, Inc. is a biotechnology company based in Carlsbad, California, that specializes in discovering and developing RNA-targeted therapeutics. The company has 3 commercially approved medicines: Spinraza (Nusinersen), Tegsedi (Inotersen), and Waylivra (Volanesorsen) and has 4 drugs in pivotal studies: tominersen for Huntington’s disease, tofersen for SOD1-ALS, AKCEA-APO(a)-LRx for cardiovascular disease, and AKCEA-TTR-LRx for all forms of TTR amyloidosis. The company was named Isis Pharmaceuticals until December 2015.

March 9, 2023


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