FDA Committee in Favor of Waylivra for Rare Metabolic Lipid Disorder

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Familial chylomicronemia syndrome is an ultra-rare genetic disease caused by impaired enzyme lipoprotein lipase.
Familial chylomicronemia syndrome is an ultra-rare genetic disease caused by impaired enzyme lipoprotein lipase.

Akcea Therapeutics announced that the Food and Drug Administration (FDA)'s Division of Metabolism and Endocrinology Products Committee voted (12-8) in favor of Waylivra's (volanesorsen) approval for the treatment of patients with familial chylomicronemia syndrome (FCS).

FCS is an ultra-rare genetic disease caused by impaired enzyme lipoprotein lipase (LPL) which prevent the breakdown of chylomicrons. Patients with FCS have severe triglyceridemia (>880mg/dL) and have an increased risk of fatal acute pancreatitis. There is currently no treatment for FCS.

Waylivra utilizes Ionis' proprietary antisense technology to lower the production the ApoC-III, a protein produced in the liver that is involved in triglyceride regulation and other metabolic parameters. Antisense technology refers to the use of synthetic nucleic acid sequences to interrupt specific protein production by targeting the corresponding messenger RNA that encodes that protein. 

The Committee reviewed findings from two Phase 3 trials (APPROACH, COMPASS) and 1 open-label study (APPROACH) for Waylivra. Data from the APPROACH trial showed that patients who received Waylivra had a statistically significant mean reduction in triglycerides of 77% from baseline as well as a reduced risk of pancreatitis. Injection site reactions and decreased platelets were the most common adverse events reported. 

The FDA is not bound to the Committee's recommendation but will be considered in its review of the New Drug Application. The FDA has set a Prescription Drug User Fee Act (PDUFA) target date for August 30, 2018.

For more information visit AkceaTx.com.

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