Ionis Pharmaceuticals, AstraZeneca Get FDA Approval for Rare Genetic Disease Treatment

By Ben Glickman

Ionis Pharmaceuticals and AstraZeneca's Wainua has been approved by the U.S. Food and Drug Administration for treating a symptom of a rare genetic disease.

The FDA approved Wainua, which has generic name eplontersen, for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

Ionis said Wainua would be available in the U.S. in January. Regulatory reviews elsewhere in the world are currently underway.

The disease, also known as hAATR, can cause polyneuropathy, a form of nerve damage. The companies said Wainua is the only approved medicine for treating polyneuropathy from hATTR.

The approval was based on a 35-week interim analysis from a Phase 3 study.

Write to Ben Glickman at ben.glickman@wsj.com

(END) Dow Jones Newswires

December 21, 2023 19:23 ET (00:23 GMT)

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