FDA Accepts Ionis NDA for Eplontersen in Rare Hereditary Disease

March 9, 2023

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.

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

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