FDA to Meet in May Regarding Potential FCS Treatment

Mathew Shanley

Last week, Akcea Therapeutics announced that the Division of Metabolism and Endocrinology Products of the U.S. Food and Drug Administration (FDA) will hold an advisory committee meeting to review data supporting the new drug application (NDA) for volanesorsen.

Akcea submitted the NDA for volanesorsen in August, and the drug is an investigational antisense therapy for the treatment of familial chlyomicronemia syndrome (FCS). It is being developed to reduce the production of ApoC-III, a protein found in the liver that plays a central role in the regulation of plasma triglycerides and has the potential to also affect other metabolic parameters.

FCS a rare genetic disease characterized by the buildup of large lipoprotein particles responsible for transporting dietary fat and cholesterol, or chlyomicronemia. Typically, the enzyme lipoprotein lipase (LPL) breaks down chylomicrons in the blood, however, in people with FCS, LPL does not function properly leading to higher triglyceride levels.

At present, there aren’t any approved effective treatments for this condition, and patients are advised to maintain a very strict, low-fat diet.

“Standard triglyceride-lowering agents are generally ineffective in lowering the extremely high triglyceride levels that cause the severe acute and chronic symptoms in people with FCS,” said Paula Soteropoulos, chief executive officer of Akcea Therapeutics in a press release. “If approved, volanesorsen would be the first therapy indicated for these patients. We look forward to reviewing the supporting data with members of the advisory committee.”

The intended patient population for volanesorsen suffers from severely high triglycerides and will be treated with the drug, either as a single agent or in combination with other triglyceride-lowering agents. Humans who do not produce apoC-III have lower levels of triglycerides and lower instances of cardiovascular disease, while humans with elevated levels of apoC-III have high triglycerides associated with multiple metabolic abnormalities, like resistance to insulin and/or metabolic syndrome.

Additionally, the prevalence of type 2 diabetes is increased in patients with elevated triglycerides, and humans with severely elevated levels of triglycerides are at risk of many serious health conditions, including pancreatitis, which can be life-threatening and require hospitalization.

The FDA granted orphan drug designation to volanesorsen in July, and has set a Prescription Drug User Fee Act (PDUFA) goal date of August 30, 2018 for the completion of its review.

Akcea is currently evaluating volanesorsen in the Phase 3 APPROACH study in patients with FCS.

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