Akcea Recognizes Landmark Year for Familial Chylomicronemia Syndrome (FCS) Community with Commemoration of 2nd Annual FCS Awareness Day
“We are very proud to recognize the second annual FCS Awareness Day, especially during an exciting time when the only treatment option for patients is available, which is giving hope to families affected by FCS around the world. With a treatment available in the EU we are already seeing the benefits for patients in terms of their health and ability to perform daily activities. We remain on track to launch WAYLIVRA in additional European countries in 2020 while we also continue to work for regulatory approval in the U.S. and additional geographies,” said
FCS is a debilitating genetic disease caused by impaired function of the enzyme lipoprotein lipase (LPL), which results in elevated triglyceride levels and significant risk and disease burden, including unpredictable and potentially fatal acute pancreatitis as well as chronic complications due to permanent organ damage.
The patient advocacy organization CholCo in
“We are so encouraged that people in
About WAYLIVRA® (volanesorsen)
With conditional marketing authorization from the
WAYLIVRA, a product of Ionis’ proprietary antisense technology, is designed to reduce the production of apoC-III, a protein that regulates plasma triglycerides and may also affect other metabolic parameters.
The European Commission’s marketing authorization of WAYLIVRA is based on results from the pivotal Phase 3 APPROACH study and the APPROACH Open Label Extension (OLE) study and is supported by results from the Phase 3 COMPASS study. Results from the APPROACH trial, the largest study ever conducted in patients with FCS, show that in comparison to placebo, treatment with WAYLIVRA reduced triglycerides 77% (-94% when compared to placebo), and reduced the incidence of pancreatitis. All patients in the trial maintained a low-fat diet.
WAYLIVRA is associated with risk of thrombocytopenia. Enhanced monitoring is required to support early detection and management of thrombocytopenia. The most frequently observed adverse reactions (more than 10%) during treatment with WAYLIVRA were events associated with injection site reactions, immunogenicity and reduction in platelet levels.
The WAYLIVRA Early Access Program (EAP) has been initiated in
FCS is an ultra-rare disease caused by impaired function of the enzyme lipoprotein lipase (LPL) and characterized by severe hypertriglyceridemia (>880mg/dL or 10mmol/L) and a risk of unpredictable and potentially fatal acute pancreatitis. Because of limited LPL production or function, people with FCS cannot breakdown chylomicrons, lipoprotein particles that are 90% triglycerides. In addition to pancreatitis, FCS patients are at risk of chronic complications due to permanent organ damage, including chronic pancreatitis and pancreatogenic (type 3c) diabetes. They can experience daily symptoms including abdominal pain, generalized fatigue and impaired cognition that affect their ability to work. People with FCS also report major emotional and psychosocial effects including anxiety, social withdrawal, depression and brain fog. Additional information on FCS is available at www.fcsfocus.com, through the LPLD Alliance at www.lpldalliance.org and through The FCS Foundation at http://www.livingwithfcs.org. For a full list of organizations supporting the FCS community worldwide, please click here.
Akcea Investor Contact:
Vice President, Corporate Communications and Investor Relations
Akcea Media Contact:
T: 212 253-8881
T: 212 253-8881
Source: Akcea Therapeutics, Inc.