Press Releases
Akcea Therapeutics Supports World Lipodystrophy Day
"We are proud to join forces with the patient community in celebration of World Lipodystrophy Day. We stand in support of all those affected by familial partial lipodystrophy (FPL). We recognize the urgent need to bring new treatments to these patients. The progress we have made with our Phase 3 drug, volanesorsen, reflects our commitment to developing and commercializing transformative therapies that address the root causes of serious, underserved cardiometabolic disorders, like FPL," said
FPL is a rare, genetic disorder characterized by metabolic abnormalities, including hypertriglyceridemia and extreme insulin resistance, and abnormalities in the distribution of body fat. Patients with FPL have a high risk at an early age of diabetes, liver disease, and cardiovascular disease. In addition, extreme hypertriglyceridemia puts them at risk for potentially life-threatening pancreatitis. Since it is most often an inherited, autosomal dominant disorder, FPL can be passed down from one generation to the next in affected families.
World Lipodystrophy Day is an international campaign designed to focus attention on the impact of living with lipodystrophy.
ABOUT VOLANESORSEN
Volanesorsen is a Gen. 2.0+ antisense drug designed to reduce the production of apoC-III, a protein that acts as a key regulator of triglyceride levels in the blood. Patients with elevated triglyceride levels are at significant risk for coronary artery disease and diabetes. Extremely high triglyceride levels put patients at risk of pancreatitis, a serious and potentially life-threating illness. In addition, elevated levels of apoC-III are recognized as an independent contributor to cardiovascular disease. Volanesorsen is currently in Phase 3 trials for two rare, genetic diseases.
ABOUT LIPODYSTROPHY UNITED
Lipodystrophy United (LU) is an organization of committed individuals living strong with lipodystrophy. LU's mission is to provide an interactive community, facilitating support and education for anyone affected by this rare disease.
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
ABOUT
Ionis is the leading company in RNA-targeted drug discovery and development focused on developing drugs for patients who have the highest unmet medical needs, such as those patients with severe and rare diseases. Using its proprietary antisense technology, Ionis has created a large pipeline of first-in-class or best-in-class drugs, with over a dozen drugs in mid- to late-stage development. Drugs currently in Phase 3 development include volanesorsen, a drug Ionis is developing and plans to commercialize through its wholly owned subsidiary, Akcea Therapeutics, to treat patients with familial chylomicronemia syndrome and familial partial lipodystrophy; IONIS-TTRRx, a drug Ionis is developing with GSK to treat patients with all forms of TTR amyloidosis; and nusinersen, a drug Ionis is developing with Biogen to treat infants and children with spinal muscular atrophy. Ionis' patents provide strong and extensive protection for its drugs and technology. Additional information about Ionis is available at www.ionispharma.com.
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SOURCE
Amy Williford, Ph.D., Associate Director, Corporate Communications, 760-603-2772