Akcea Therapeutics, Inc. announced that the German Association of Statutory Sick Funds has agreed upon new pricing and reimbursement of WAYLIVRA (volanesorsen) 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 has been commercially available in Germany since August 15, 2019. As part of reimbursement and market access protocol in Germany, Akcea was required to renegotiate the WAYLIVRA launch price 12 months after first commercialization, which will go into effect on August 15, 2020. 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. It is estimated that there are between 3,000 to 5,000 people living with FCS worldwide. WAYLIVRA is designed to reduce the production of apolipoprotein C-III (apoC-III), a protein produced in the liver that plays a central role in the regulation of plasma triglycerides. WAYLIVRA (volanesorsen): With conditional marketing authorization from the European Commission (EC) as an adjunct to diet in adult patients with genetically confirmed FCS and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate, WAYLIVRA is now the only therapy indicated for people with familial chylomicronemia syndrome (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 Phase 3 APPROACH study and the ongoing APPROACH Open Label Extension (OLE) study and is supported by results from the Phase 3 COMPASS study. Results from the Phase 3 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). 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. 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.