ICYMI: Successful Phase 3 Study of Familial Chylomicronemia Syndrome Drug May Win Drug Approval in US

According to a publication from BioPortfolio, American biotechnology companies Akcea Therapeutics and Ionis Pharmaceuticals recently published final study results from their phase 3 clinical study of Waylivra (generic name volanesorsen) in familial chylomicronemia syndrome patients. The results were published earlier this month in an issue of the prestigious New England Journal of Medicine.

About Familial Chylomicronemia Syndrome

Familial chylomicronemia syndrome (FCS) is a rare, genetically inherited condition characterized by the accumulation of abnormally high levels of triglycerides (hypertriglyceridemia) in the blood and plasma.

Triglycerides are a type of fat molecule — in fact, they are the most abundant type of fat in the human body. Having high triglyceride levels can have serious health consequences, including heart disease, coronary artery disease, and, especially in FCS patients — sometimes fatal episodes of acute pancreatitis.

In FCS patients, the characteristic hypertriglyceridemia is most often caused by a genetic mutation that leads to the underproduction of an enzyme called lipoprotein lipase (LPL). In otherwise healthy individuals, LPL breaks down lipoproteins called chylomicrons, which are made up almost entirely of triglycerides.

The result is often dangerously high blood triglyceride levels, which can cause permanent organ damage, and even early mortality in some cases. While no treatment for the condition has yet been approved for market in the United States, Akcea Therapeutics and Ionis Pharmaceuticals’ Waylivra (volanesorsen) was approved in Europe earlier this year.

Volanesorsen Shows Promise in Phase 3 Study

Akcea Therapeutics and Ionis Pharmaceuticals’ recently published phase 3 study of volanesorsen was the largest study of FCS patient cases in history. Over the course of three months, 66 FCS patients from a dozen different countries received either volanesorsen or a placebo. Since only an estimated three-to-five thousand people in the world have FCS, a participant group of this size is especially impressive. To eliminate any potential bias at the time of the experiment, neither the participants nor the administering researchers knew which participants were in the control or active groups.

After three months of observation, researchers were pleased with their findings. Overall, the active group achieved an average reduction in triglyceride levels of 77%. The group also enjoyed an average reduction of 84% in apolipoprotein C-III levels. Apolipoprotein C-III is a protein produced in the liver that is closely linked to plasma triglyceride regulation.

Overall, triglyceride levels were reduced to “…below the threshold for risk of triglyceride-induced acute pancreatitis,” according to Dr. Marcello Arca of Policlinico Umberto I hospital in Rome.

The success of this study may finally pave the way for volanesorsen’s approval in the United States.

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