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

Xconomy San Diego — 

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 FDA rejected the drug.

The news, which was released after markets closed Monday, comes hours after Pfizer (NYSE: PFE) gained momentum as a potential competitor to Ionis and Akcea in a different disease area, transthyretin amyloidosis (ATTR). Their ATTR drug, inotersen, is based on the same biological mechanism as volanesorsen; it intercepts and destroys the genetic code that a patient’s cells would otherwise use to produce a disease-causing protein.

Inotersen is approved in Europe and faces an FDA decision this fall. As Xconomy reported this summer, inotersen has a spottier safety record than its competitors. It caused the same serious side effect, thrombocytopenia, that also dogged volanersorsen. Ionis shares are down 16 percent in aftermarket trading. Akcea shares are down 28 percent.

Image courtesy of Juhan Sonin via Creative Commons. It has been modified from the original source.