Ionis, AstraZeneca Get FDA Orphan Designation for Eplontersen
25 January 2022 - 12:09AM
Dow Jones News
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 colin.kellaher@wsj.com
(END) Dow Jones Newswires
January 24, 2022 07:54 ET (12:54 GMT)
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