Akcea Therapeutics’ volanesorsen has taken a step closer to becoming the first therapy on the US market for the ultra-rare rare lipid disorder familial chylomicronemia syndrome (FCS).
The US Food and Drug Administration’s Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) has voted 12-8 in favor of approving the drug, which is to be marketed under the brand name Waylivra.
FCS is a severe, potentially fatal disorder where patients have extremely high levels of triglycerides in their blood, causing a range of symptoms including potentially fatal attacks of acute pancreatitis.
People with the condition are unable to metabolise large triglyceride-rich lipid particles called chylomicrons due to a deficiency in lipoprotein lipase, an enzyme that helps to break down triglycerides.
Currently, the only option for patients is a severely restricted low fat diet of 10-20 grams of fat a day, which, even when strictly adhered to, does not decrease triglycerides to normal levels or remove the threat of pancreatitis for most patients, a company spokesperson noted.
Paula Soteropoulos, chief executive officer of Akcea, said the submission includes data from three clinical trials, which “demonstrate clear improvement on several important measures of disease in these patients”.
Volanesorsen is also under review in Europe, but is already available to FCS patients in the UK under the Medicines and Healthcare products Regulatory Agency’s Early Access to Medicines Scheme.
The FDA should make a decision on drug by August 30.