New England Journal of Medicine Publishes Results from Pivotal Study of WAYLIVRA® (volanesorsen) in Patients with Familial Chylomicronemia Syndrome
Mean triglyceride levels decreased 77% in volanesorsen-treated patients versus an 18% increase in patients in placebo group
Akcea launching WAYLIVRA in the European Union
BOSTON and CARLSBAD, Calif, Aug. 07, 2019 (GLOBE NEWSWIRE) -- Akcea Therapeutics, Inc. (NASDAQ: AKCA), an affiliate of Ionis Pharmaceuticals, Inc., and Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), announced today that the final study results from the Phase 3 APPROACH study evaluating WAYLIVRA® (volanesorsen) in patients with familial chylomicronemia syndrome (FCS) were published in the August 8th issue of The New England Journal of Medicine (NEJM). The manuscript is titled “Volanesorsen and Triglyceride Levels in Familial Chylomicronemia Syndrome.”
FCS is an ultra-rare debilitating disease that can be life-altering. It is caused by impaired production or function of the enzyme, lipoprotein lipase (LPL), responsible for breaking down chylomicrons, lipoproteins rich in triglycerides. This results in extremely high triglyceride levels that lead to 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.
“Results from the APPROACH study indicate that WAYLIVRA lowers triglyceride levels below the threshold for risk of triglyceride-induced acute pancreatitis in the majority of patients,” said Dr. Marcello Arca, Head of the Lipid and Atherosclerosis Unit of the University Hospital Policlinico Umberto I. “Acute pancreatitis is one of the most severe consequences of FCS, causing hospitalization, missed work or school, reduced time with family in addition to the potential for permanent organ damage or death. WAYLIVRA is a promising treatment that could significantly reduce the burden of FCS for patients and their families.”
APPROACH was a Phase 3, double-blind, randomized, placebo-controlled clinical trial designed to evaluate the safety and efficacy of WAYLIVRA in FCS patients. It is the largest study ever conducted in FCS to-date, involving 66 patients from 40 different treatment centers in 12 countries. Results from the APPROACH trial show that FCS patients treated with WAYLIVRA achieved a statistically significant mean reduction in triglycerides of 77 percent from baseline (mean reduction of 94 percent when compared to placebo) at three months. The levels of apoC-III also decreased by an average of 84 percent from baseline at three months (P<0.001) following treatment with WAYLIVRA. The most common adverse events were injection site reactions and platelet declines. For the full text of this publication, please visit: https://www.nejm.org/doi/full/10.1056/NEJMoa1715944?query=featured_home.
“We are very pleased that The New England Journal of Medicine recognizes the significance of the APPROACH data and the potential of WAYLIVRA to address an area of significant unmet need in the treatment of FCS,” said Louis O’Dea, chief medical officer of Akcea Therapeutics. “WAYLIVRA is the only treatment approved for FCS. We are currently preparing to make WAYLIVRA available to patients in Europe beginning in Germany this month. Our hope is that we will be able to deliver WAYLIVRA to patients with FCS in other geographies who desperately need an approved treatment.”
“This publication of the APPROACH study results the recent EU approval of WAYLIVRA, clearly demonstrates WAYLIVRA’s safety and efficacy profile for patients with FCS. We and Akcea are focusing on delivering this much needed medicine to those patients living with this severe disease who are waiting to have WAYLIVRA as a treatment option,” said Brett P. Monia, Ph.D., chief operating officer of Ionis Pharmaceuticals.
About WAYLIVRA® (volanesorsen)
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 fro 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.
The WAYLIVRA Early Access Program (EAP) has been initiated in Europe, the U.S. 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.
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