According to a story from cafepharma.com, Akcea Therapeutics, Inc. recently announced that the FDA’s Division of Metabolism and Endocrinology Products Advisory Committee voted to recommend approval of the therapy Waylivra (volanesorsen). The drug was approved in a 12-8 votes. Waylivra is designed for the treatment of familial chylomicronemia syndrome.
Familial chylomicronemia syndrome (FCS), also known as lipoprotein lipase deficiency is a genetic disorder caused by a mutation. This results in a deficiency in the production of lipoprotein lipase enzymes, which play a major role in the breakdown of triglycerides. As a result, triglycerides appear at excessively high levels in the blood (hypertriglyceridemia). This accumulation of these fatty molecules can cause a blood sample to have a creamy appearance. Disease effects are present early in life, and infants may display overall failure to thrive and pain resembling colic. The most common symptoms of familial chylomicronemia syndrome throughout a patient’s life are acute pancreatitis and abdominal pain. Acute pancreatitis can lead to serious complications, such as pancreatic insufficiency and diabetes. Management involves the use of lipid lowering drugs and adherence to a strict diet low in fats and simple carbs. To learn more about familial chylomicronemia syndrome, click here.
There is a significant medical need for more treatment options for patients with FCS. In some patients, the conventional method of dieting and lipid drugs is not enough for them to avoid symptoms and dangerous complications. Waylivra has the potential to provide a back up option when the conventional treatment methods are no sufficient.