Familial chylomicronemia Syndrome (FCS) is a genetic disorder characterized by impaired function of the enzyme lipoprotein lipase (LPL), leading to the inability to break down chylomicrons and the buildup of chylomicrons in the blood. Chylomicrons are lipoprotein particles primarily made up of triglycerides. This buildup results in a significant increase in triglyceride levels, severe abdominal pain, and potentially fatal episodes of pancreatitis. Currently, there are no approved drug therapies to treat FCS. Patients are managed with dietary restrictions such as reductions in the consumption of dietary fat.
Volanesorsen (Waylivra TM), an investigational antisense lipid-modifying agent, is being developed by Akcea Therapeutics in collaboration with Ionis Pharmaceuticals for the treatment of familial chylomicronemia syndrome (FCS). Volanesorsen received Orphan Drug Designation in June of 2015 for its familial chylomicronemia syndrome (FCS) indication and has a current PDUFA action date of August 30, 2018.
For our complete review of Volanesorsen sodium:
Executive Summary of Evaluation:
Volanesorsen (Waylivra TM)
(Akcea Therapeutics / Ionis Pharmaceuticals)
|Orphan Drug Designation||
|Indications||Familial Chylomicronemia Syndrome (FCS)||
Submitted for Approval (NDA)
|Efficacy||Familial Chylomicronemia Syndrome (FCS)||
Efficacy reported in Phase III Trials
“About FCS”. The FCS Foundation. https://www.livingwithfcs.org/What-is-FCS/About-FCS . Accessed: July 12, 2018.
Clinical Trial data obtained at https://clinicaltrials.gov/ct2/home Accessed: July 9, 2018.
“FDA Briefing Document: Endocrinologic and Metabolic Drugs Advisory Committee Meeting, May 10, 2018”. U.S. Food and Drug Administration. https://www.fda.gov/…/EndocrinologicandMetabolicDrugsAdvisoryCommittee/UCM606857.pdf. Accessed: July 16, 2018.
Gaudet D, Digenio A, Alexander VJ, Arca M, Jones AF, et al. The APPROACH Study: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of Volanesorsen Administered Subcutaneously to Patients with Familial Chylomicronemia Syndrome (FCS). Journal of Clinical Lipidology. 2017 May;11(3):814-15.
Gouni-Berthold I, Alexander V, Digenio A, DuFour R, Steinhagen-Thiessen E, et al. Apolipoprotein C-III Inhibition With Volanesorsen in Patients With Hypertriglyceridemia (COMPASS): A Randomized, Double-Blind, Placebo-Controlled Trial. Journal of Clinical Lipidology. 2017 May;11(3):794-95.
Global Data, Pharma Intelligence Center https://pharma.globaldata.com Accessed: July 25, 2018.
“Understanding Familial Chylomicronemia Syndrome”. Rare Disease Report. http://fcs.raredr.com. Accessed: July 17, 2018.
Additional 2018 Pipeline Reviews are available for the following drugs.
Coauthored by: Kristen Ciampi, PharmD
Kristen recently graduated with a PharmD degree from the University of Rhode Island in May of 2018, and is a Pharmacy Analytics Intern at RJ Health Systems.