From Dow Jones & Company:



The FDA has approved Wainua, developed by Ionis Pharmaceuticals and AstraZeneca, for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. This rare genetic disease can cause nerve damage, and Wainua is the only approved medicine for treating this specific symptom. The drug will be available in the U.S. in January, with regulatory reviews underway in other parts of the world. The approval was based on a 35-week interim analysis from a Phase 3 study.



Original: Ionis, AstraZeneca get FDA approval for rare genetic disease treatment