Approval and launch of WAYLIVRA® (volanesorsen) in Europe, the only treatment option for FCS patients, highlights progress in clinical research
BOSTON, Nov. 01, 2019 (GLOBE NEWSWIRE) -- Akcea Therapeutics, Inc. (AKCA), a majority-owned affiliate of Ionis Pharmaceuticals, Inc. (IONS), today recognizes Familial Chylomicronemia Syndrome (FCS) Awareness Day. FCS Awareness Day is a global observance held each year on the first Friday in November to build broader awareness of the challenges associated with this devastating, ultra-rare disease and the critical need for treatment. Along with members of the FCS community around the world, Akcea celebrates the significant progress made over the past year, including the EU approval of WAYLIVRA, the only treatment approved for FCS, as an adjunct to diet in adult patients with genetically confirmed FCS and at high risk for pancreatitis.
“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 Damien McDevitt, Ph.D., interim chief executive officer at Akcea.
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 Germany also issued a statement: “The FCS community in Europe, including patients, their caregivers and their families, is thankful that research has led to a treatment option for this devastating disease. With access to treatment, EU patients have the potential to reduce their triglyceride levels, which has been shown to correlate with reduced risk of abdominal pain and potentially fatal attacks of pancreatitis.”
“We are so encouraged that people in Europe living with FCS now have a treatment available. This milestone brings new levels of hope and dedication to patients in the U.S., Canada and other countries around the world who are still waiting for a treatment,” said Lindsey Sutton, co-founder of the FCS Foundation and a patient living with FCS. “The FCS community is motivated now more than ever to build on a year of amazing momentum and continue our fight for a treatment to be available to all patients with FCS no matter where they live.”
About WAYLIVRA® (volanesorsen)
With conditional marketing authorization from the European Commission (EC) in May 2019 as an adjunct to diet in adult patients with genetically confirmed familial chylomicronemia syndrome (FCS) and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate, WAYLIVRA is now the only approved therapy for people with FCS. Akcea is working to confirm a path forward for WAYLIVRA in the U.S. and Canada.
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 Europe and Canada and is currently enrolling eligible patients. Click here for more information on the WAYLIVRA EAP. For more information on WAYLIVRA, please visit www.WAYLIVRA.eu.
In August 2019 Akcea and Ionis announced positive top-line results for the BROADEN study of WAYLIVRA for the treatment of patients with familial partial lipodystrophy, or FPL. To view the release, click here.
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.
ABOUT AKCEA THERAPEUTICS, INC.
Akcea Therapeutics, Inc., an affiliate of Ionis Pharmaceuticals, Inc., is a biopharmaceutical company focused on developing and commercializing drugs to treat patients with serious and rare diseases. Akcea is commercializing TEGSEDI® (inotersen) and WAYLIVRA® (volanesorsen) as well as advancing a mature pipeline of novel drugs, including AKCEA-APO(a)-LRx, AKCEA-ANGPTL3-LRx, AKCEA-APOCIII-LRx, and AKCEA-TTR-LRx, with the potential to treat multiple diseases. All six drugs were discovered by Ionis, a leader in antisense therapeutics, and are based on Ionis’ proprietary antisense technology. TEGSEDI is approved in the U.S., E.U. and Canada. WAYLIVRA is approved in the E.U. and is currently in Phase 3 clinical development for the treatment of people with familial partial lipodystrophy, or FPL. Akcea is building the infrastructure to commercialize its drugs globally. Akcea is a global company headquartered in Boston, Massachusetts. Additional information about Akcea is available at www.akceatx.com and you can follow us on twitter at @akceatx.
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