Waylivra gets conditional EU nod in ultra rare disease

While Waylivra (volanesorsen) has been snubbed by the US Food and Drug Administration, Europe’s regulator saw enough hopeful data to give the therapy a conditional approval in familial chylomicronemia syndrome (FCS).

This condition is a debilitating ultra rare disease caused by impaired function of the enzyme lipoprotein lipase, which causes high circulating levels of triglycerides in the body, leading to extreme abdominal pain and episodes of potentially fatal pancreatitis.

Until now, there has been no treatment available for patients, whose only option to manage their disease was to follow an extreme low-fat diet of less than 10-15g of fat a day – the equivalent to two thirds of an avocado.