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Ionis and AstraZeneca obtain FDA approval for rare genetic disease treatment
IONIS Pharmaceuticals (IONS)
AstraZeneca’s AZN;
Wainua is approved by the U.S. Food and Drug Administration (FDA) to treat symptoms of a rare genetic disorder.
The FDA has approved Wainua, with the generic name eplontersen, for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
Ionis said Wainua will launch in the U.S. in January. A regulatory review is currently underway globally.
This disease, also known as hAATR, can cause polyneuropathy, a form of nerve damage. The companies said Whynua is the only drug approved for the treatment of hATTR polyneuropathy.
This approval was based on the 35-week interim analysis of the Phase 3 study.
