Ionis/AstraZeneca's eplontersen an Orphan Drug in U.S. for transthyretin-mediated amyloidosis
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Ionis Pharmaceuticals (NASDAQ:IONS)
announces that the FDA has granted Orphan Drug Designation to eplontersen, an investigational antisense medicine for the treatment of people living with transthyretin-mediated amyloidosis, a systemic, progressive and fatal condition.
Among the benefits of Orphan Drug status in the U.S. is a seven-year period of market exclusivity for the indication, if approved.

Transthyretin amyloidosis is a slowly progressive condition characterized by the buildup of abnormal deposits of a protein called amyloid (amyloidosis) in the body's organs and tissues.
In December 2021, Ionis collaborated with AstraZeneca (NASDAQ:AZN) to develop and commercialize eplontersen.