Ionis, AstraZeneca Get FDA Orphan Designation for Eplontersen

By Colin Kellaher

Ionis Pharmaceuticals Inc. and AstraZeneca PLC on Monday said the U.S. Food and Drug Administration granted orphan-drug designation to their investigational antisense drug eplontersen for the treatment of transthyretin-mediated amyloidosis, a systemic, progressive and fatal condition.

The companies said eplontersen, currently in Phase III studies for amyloid transthyretin cardiomyopathy and amyloid transthyretin polyneuropathy, is designed to reduce the production of transthyretin to treat both hereditary and nonhereditary forms of transthyretin-mediated amyloidosis.

The FDA's orphan-drug program gives special status to drugs and biologics for diseases and disorders that affect fewer than 200,000 people in the U.S. and provides for an extended marketing exclusivity period against competition.

Ionis, a Carlsbad, Calif., pharmaceutical company, and Anglo-Swedish drugmaker AstraZeneca in December entered into a global development and commercialization agreement for eplontersen.

The companies said they expect hereditary amyloid transthyretin polyneuropathy will be the first indication for which they will seek regulatory approval for eplontersen, with a potential filing with the FDA by the end of the year.

Write to Colin Kellaher at [e-mail]

(END) Dow Jones Newswires

January 24, 2022 07:54 ET (12:54 GMT)

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