AstraZeneca (AZN.L,AZN) reported that Eplontersen has been granted Orphan Drug Designation in the US for the treatment of transthyretin-mediated amyloidosis. Eplontersen is a ligand-conjugated antisense investigational medicine currently in phase III clinical trials for amyloid transthyretin cardiomyopathy and amyloid
transthyretin polyneuropathy.
eplontersen will be jointly developed and commercialized by Ionis and AstraZeneca in the US. 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 FDA by the end of 2022.
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