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2022 | HFSA

Eplontersen granted Orphan Drug Designation in the US for transthyretin amyloidosis

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WILMINGTON, Del., January 24, 2022 – Eplontersen has been granted Orphan Drug Designation (ODD) in the US by the Food and Drug Administration (FDA) for the treatment of transthyretin-mediated amyloidosis, a systemic, progressive and fatal condition.

Eplontersen, formerly known as IONIS-TTR-LRx, is a ligand-conjugated antisense (LICA) investigational medicine currently in Phase III 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).

ATTR-CM is a systemic, progressive and fatal condition that leads to progressive heart failure and death within four years from diagnosis.1 It remains underdiagnosed and its prevalence is thought to be underestimated due to a lack of disease awareness and the heterogeneity of symptoms.2,3 Hereditary ATTR-PN is a debilitating disease that leads to peripheral nerve damage with motor disability within five years of diagnosis and, without treatment, is generally fatal within a decade.3

The FDA grants ODD status to medicines and potential new medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US.

Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, AstraZeneca, said: “Eplontersen has the potential to be a best-in-class treatment to halt the progression of transthyretin-mediated amyloidosis and treat this fatal condition. The FDA designation further underscores the potential for eplontersen to offer new hope to this patient population currently faced with limited treatment options.”

As part of a global development and commercialization agreement with Ionis Pharmaceuticals, Inc. (Ionis) eplontersen will be jointly developed and commercialized by both companies in the US, and will be developed and commercialized in the rest of the world, except in Latin America, by AstraZeneca.

Hereditary ATTR-PN is expected to be the first indication for which the companies will seek regulatory approval for eplontersen, with the potential to file a new drug application with the US Food and Drug Administration by the end of 2022.