"FCS is a rare and very serious genetic disorder associated with very high triglyceride levels that put patients at risk of potentially life-threatening pancreatitis. People with FCS cope with daily consequences of their disease, including persistent abdominal pain and life-altering food restrictions. Unfortunately, current treatment options do not reduce triglyceride levels enough to reduce the risk of serious illness in patients with FCS," said
The Orphan Drug Act provides for economic incentives to encourage the development of drugs for diseases affecting fewer than 200,000 people in the United States. Orphan drug designation entitles Akcea Therapeutics to seven years of market exclusivity in
FCS is a rare genetic disorder characterized by extremely high levels of triglycerides that affects an estimated one to two out of a million people. FCS may also be called familial chylomicronemia, Frederickson Type I hyperlipoproteinemia, familial lipoprotein lipase deficiency (LPLD), or familial hypertryglyceridemia. Some people with FCS may live with recurrent stomach pain, and a high risk or prior history of hospitalization for pancreatitis. FCS is primarily caused by mutations in genes that produce proteins involved in the clearance of particles that carry triglycerides, called chylomicrons. Patients with FCS are unable to effectively clear chylomicrons, and as such, have high levels of triglycerides, which increase their risk of pancreatitis, type 2 diabetes and other serious illnesses.
Volanesorsen (ISIS-APOCIIIRx) is an antisense drug in development intended to treat patients with severely high triglycerides either as a single agent or in combination with other triglyceride-lowering agents. Volanesorsen is designed to target apoC-III, a protein produced in the liver that plays a central role in the regulation of serum triglycerides.2 Humans who do not produce apoC-III have lower levels of triglycerides and lower instances of cardiovascular disease.3 Humans with elevated levels of apoC-III have high triglycerides associated with multiple metabolic abnormalities, such as insulin resistance and/or metabolic syndrome.4 In addition, the prevalence of type 2 diabetes is increased in patients with elevated triglycerides.5 Humans with severely elevated levels of triglycerides are at risk of many serious health conditions, including pancreatitis,4 which can be life-threatening and require hospitalization. Volanesorsen is currently being evaluated in a Phase 3 study in patients with FCS. A second Phase 3 study of volanesorsen is planned to begin later this year in patients with Familial Partial Lipodystrophy, another severe and rare lipid disorder. For more information about this clinical trial program, please visit www.apociii.com
ABOUT AKCEA THERAPEUTICS
Akcea Therapeutics is a development and commercialization company focused on transforming the lives of patients with serious cardiometabolic lipid disorders. Established as a wholly owned subsidiary of
Isis is exploiting its leadership position in RNA-targeted technology to discover and develop novel drugs for its product pipeline and for its partners. Isis' broad pipeline consists of 38 drugs to treat a wide variety of diseases with an emphasis on cardiovascular, metabolic, severe and rare diseases, including neurological disorders, and cancer. Isis' partner, Genzyme, is commercializing Isis' lead product, KYNAMRO®, in
This press release includes forward-looking statements regarding the business of
In this press release, unless the context requires otherwise, "Akcea," "Company," "we," "our," and "us" refers to Akcea Therapeutics.
Isis Pharmaceuticals® is a registered trademark of
- Gaudet, D. et al. (2014). Targeting APOC3 in the familial chylomicronemia syndrome. N Engl J Med, 374(23), 2200-2206.
- Zheng, C. (2014). Updates on apolipoprotein CIII: fulfilling promise as a therapeutic target for hypertriglyceridemia and cardiovascular disease. Curr Opin Lipidol, 25(1), 35-39.
Jorgensen, A.B., Frikke-Schmidt, R., Nordestgaard, B.G. & Tybaerg-Hansen, A. (2014) Loss-of-function mutations in APOC3 and risk of ischemic vascular disease. N Engl J Med, 371(1), 32-41
- Christian, J.B., Arondekar, B., Buysman, E.K., Jacobson, T.A., Snipes, R.G., Horwitz, R. (2014). Determining triglyceride reductions needed for clinical impact in severe hypertriglyceridemia. Am J Med, 127(1), 36-44.
- Mooradian, A.D. (2009). Dyslipidemia in type 2 diabetes mellitus. Nat Clin Pract Endocrinol Metab, 5(3), 150-159.
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D. Wade Walke, Ph.D., Vice President, Corporate Communications and Investor Relations, 760-603-2741, OR Amy Williford, Ph.D., Associate Director, Corporate Communications, 760-603-2772