According to a story from Akcea Therapeutics, the company recently received a reply from the US Food and Drug Administration (FDA) in regards to their New Drug Application for WAYLIVRA (volanesorsen). Disappointingly, the agency opted to reject the drug for market approval. For a disease with limited treatment options, both Akcea and the patient community expressed their dismay at the news.
About Familial Chylomicronemia Syndrome
Familial chylomicronemia syndrome, which is also known as lipoprotein lipase deficiency, is a rare genetic disorder which is characterized by the inadequate production of lipoprotein lipase due to a defective gene. This results in very high levels of triglycerides. The signs and symptoms of the condition include fat deposits under the skin and stomach pain. Without intervention, the disorder can lead to liver problems and pancreatitis. This can also eventually cause the patient to develop diabetes. When the syndrome first appears, it often presents with failure to thrive and colicky pain. Treatment for familial chylomicronemia syndrome often involves a strict diet that is low in fat and simple carbs. Lipid lowering drugs can also be of some use. In the EU, there is a gene therapy available for patients who experience severe symptoms despite diet restrictions, but only one patient has ever used it. To learn more about familial chylomicronemia syndrome, click here.
A Letdown For Patients
In response to the news, the Facebook page Living with Familial Chylomicronemia Syndrome released a post highlighting why this new drug would have been a valuable asset to patients:
“The FCS Community is very disappointed to get news of this decision from the FDA regarding Waylivra, the only drug suitable for helping individuals manage pain and symptoms of FCS…”
There are currently no real treatment alternatives for those patients who are not able to manage their syndrome through dieting in the US. It is not entirely clear what led the FDA to decide to reject the drug.
Promising in Trials
In trials, Waylivra was able bring down triglyceride levels by 77 percent. Bringing down triglyceride levels is a primary goal of managing familial chylomicronemia syndrome, as it can prevent symptoms and pain from appearing. The drug also did not appear to have any dangerous side effects; the most common were injection site reactions and reduction in platelet levels.