FDA Gives Thumbs-Down to Rare Disease Drug from Ionis, Akcea

The FDA has rejected the rare-disease drug volanesorsen (Waylivra), which is being developed by Ionis Pharmaceuticals (NASDAQ: IONS) and its subsidiary Akcea Therapeutics (NASDAQ: AKCA). Ionis, based in San Diego, and Akcea, in Cambridge, MA, had high hopes for approval after a committee of outside experts in May recommended the drug by a 12-8 vote. A majority of advisers voted for volanesorsen, a treatment for familial chylomicronemia syndrome, or FCS, despite safety concerns. FCS causes a painful, often damaging buildup of fat in a patient’s organs. In Monday’s announcement, Ionis and Akcea did not say why the… Read more »

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