According to a story from BioSpace, the U.S. Food and Drug Administration (FDA) recently rejected the approval of the drug Waylivra, which was in development for the treatment of familial chylomicronemia syndrome. The rejection came as shock to many in the disease community since the drug had performed well in trials. Now, the Familial Chylomicronemia Foundation is pressuring the agency to reconsider its decision.
About Familial Chylomicronemia Syndrome
Familial chylomicronemia syndrome, which is also known as lipoprotein lipase deficiency, is a genetic disorder in which a certain enzyme is deficient, which causes abnormally high levels of triglycerides to build up in the patient’s body. The can result in serious health complications. Symptoms of familial chylomicronemia syndrome include deposits of fat under the skin, severe abdominal pain, and complications of the liver and pancreas, such as pancreatitis. This can result in pancreatic insufficiency which can lead to diabetes. The main treatment approach is a diet that avoids simple carbs and fat. Currently available lipid lowering drugs are only marginally useful. There is also a gene therapy drug approved in Europe for patients with acute pancreatitis despite dieting measures, but only one person has ever used it since its approval. To learn more about familial chylomicronemia syndrome, click here.
A Confusing Decision
The reasoning behind the FDA’s decision to reject the drug is not entirely clear. In trials the therapy was able to drop lipid levels by 77 percent in patients with familial chylmicronemia syndrome, and it did not have serious side effects. The FDA Advisory Panel also recommended that Waylivra be cleared for approval after a vote in May in 2018. During this meeting, multiple participants in clinical trials for the drug attested to its effectiveness and safety.
Patients Left Behind
The apparent lack of explanation from the agency, along with its willingness to ignore patient testimony and feedback regarding the therapy, has understandably angered many patients, as there are currently no approved drugs for the syndrome. Even with the strict diet, some patients are still susceptible to experiencing pain and other symptoms.
“The FDA should let patients, families, and their physicians make the decision on the risks and benefits of this important therapy.” – Matt Alsante, Director of the National Pancreas Foundation.