Treatment for Familial Chylomicronemia Syndrome Gets Recommendation From FDA Committee

According to a story from, 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.

This approval was based on data obtained from Phase III clinical trials that measured the effectiveness and safety of Waylivra. The results were highly encouraging, and patients in the trial experienced an average of 77 percent reduction in triglyceride levels. The risk of pancreatitis also decreased significantly.
Waylivra had previously received Orphan Drug Designation in both the U.S. and the E.U. This designation is reserved for treatments that would meet a currently unmet medical need, are geared for treating a disease considered rare, and have indicated in testing to provide an increased medical benefit for patients compared to currently available treatments. Overall, it seems clear that this drug is well on its way to seeing approval from the FDA for public use.

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