Volanesorsen for chylomicronemia

July 5, 2019 0 By FM

Akcea has been granted a conditional approval for volanesorsen (Waylivra), its antisense drug for the rare inherited disorder familial chylomicronemia syndrome (FCS), in Europe.

The European Commission has cleared volanesorsen as an adjunct to diet in adult patients with confirmed FCS who are at high risk for pancreatitis and have not responded well enough to diet and triglyceride lowering therapy. The EU is the first market for the new drug.

The US FDA turned down the company’s marketing application for volanesorsen last August. The FDA had voiced concerns about the drug’s safety, particularly some reductions in blood platelet counts among patients treated with the antisense agent.

FCS prevents the body from breaking down fats, and results in extremely high levels of triglycerides in the blood. This causes a range of symptoms including severe abdominal pain, potentially fatal attacks of acute pancreatitis, hepatosplenomegaly, diabetes, lack of concentration, memory loss and fat-filled spots on the skin.

Currently, the only option for patients is a severely restricted low-fat diet of 10-20 grams of fat a day which often fails to remove the threat of pancreatitis.

The company plans to launch the drug into a market before the end of the year with additional launches in 2020, according to reports.