Akcea and Ionis Announce Submission of Marketing Authorization Application for Volanesorsen to the European Medicines Agency
- Category: DNA RNA and Cells
- Published on Thursday, 27 July 2017 20:14
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CAMBRIDGE, MA and CARLSBAD, CA, USA I July 27, 2017 I Akcea Therapeutics, Inc. (NASDAQ: AKCA) and Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that a marketing authorization application (MAA) has been submitted to the European Medicines Agency (EMA) for volanesorsen for the treatment of patients with familial chylomicronemia syndrome (FCS). Volanesorsen, a product of Ionis' proprietary antisense technology, was discovered by Ionis and co-developed by Ionis and Akcea and, if approved, will be commercialized by Akcea.
"The submission of the volanesorsen MAA to the EMA represents a critical milestone for Akcea. This filing brings us one step closer to our goal of providing physicians and patients with a treatment for a disease with multiple, severe, daily and chronic manifestations," said Paula Soteropoulos, president and chief executive officer of Akcea. "We have had productive communications with regulatory agencies in the EU, the U.S., and Canada, and are on track to also complete regulatory submissions in the U.S. and Canada in September. Further, Akcea is building the organization and infrastructure to commercialize volanesorsen globally."
FCS is a severe, rare disorder characterized by extremely high levels of triglycerides and the risk of recurrent, potentially fatal pancreatitis. People with FCS are unable to effectively metabolize large, triglyceride-rich lipid particles called chylomicrons due to a deficiency in lipoprotein lipase, an enzyme that helps to break down triglycerides. There is no effective therapy available.
"Patients with FCS need new therapies that can meaningfully lower their severely elevated triglycerides. Because of their very high triglycerides, these patients suffer from daily and chronic symptoms and are at significantly increased risk for morbidity and mortality, primarily due to recurrent attacks of pancreatitis," said Dr. Marcello Arca, Head of the Lipid and Atherosclerosis Unit of the University Hospital Policlinico Umberto I. "Based on the positive results from the Phase 3 program with volanesorsen, I'm encouraged that for the first time, these patients may have a new therapy that can help them achieve the triglyceride reductions they need to lessen the burden of their disease and decrease their risk for pancreatitis."
"Akcea has shown great commitment in supporting patients and working to find a treatment for this debilitating condition. Their work means that the European FCS community of patients, families, and physicians are one step closer to having an effective treatment that will make the life-long struggle of living with the burden of FCS much more manageable. It is great to be working with a supportive partner like Akcea, and we also appreciate the perseverance of our community in the quest to find an effective treatment," said Jill Prawer, Founder and Chair, LPLD Alliance.
ABOUT THE VOLANESORSEN CLINICAL PROGRAM
The COMPASS study, a six-month randomized placebo-controlled study in 113 patients with very high triglycerides (>500 mg/dL), also achieved its primary endpoint of reduction in triglycerides at three months, with a 71% mean reduction in triglycerides. In the COMPASS study, treatment with volanesorsen was associated with a statistically significant reduction in on-study pancreatitis attacks.
The most common adverse event in the studies was injection site reactions, which were mostly mild. Platelet count reductions were observed in many patients. These platelet declines were not clinically significant in most patients and were generally well managed with monitoring and dose adjustment. Five patients discontinued participation in the APPROACH study due to platelet count reductions, two of which were severe; four patients discontinued due to other nonserious adverse events.
Akcea and Ionis continue to conduct the BROADEN study, a Phase 3 clinical trial in patients with FPL, which continues to enroll, with topline data expected in 2019. Akcea plans to file for marketing authorization for volanesorsen to treat FPL in 2019 if the data from the BROADEN study are positive. The U.S. and EU regulatory agencies have granted Orphan Drug Designation to volanesorsen for the treatment of patients with FCS. Volanesorsen has also received Orphan Drug Designation in the EU for the treatment of FPL.
ABOUT VOLANESORSEN, FCS AND FPL
FCS is a severe, rare disorder characterized by extremely high levels of triglycerides and the risk of recurrent, potentially fatal pancreatitis. People with FCS are unable to effectively metabolize large, triglyceride-rich lipid particles called chylomicrons due to a deficiency in lipoprotein lipase, an enzyme that helps to break down triglycerides. There is no effective therapy available. Additional information on FCS is available at www.fcsfocus.com and through the FCS Foundation at http://www.livingwithfcs.org and the LPLD Alliance at www.lpldalliance.org.
FPL is a severe, rare genetic metabolic disorder characterized by an inability of the body to store fat in normal locations. This results in high levels of triglycerides in the bloodstream, abnormal fat distribution around and within organs, such as the liver and heart, and a range of metabolic abnormalities, including severe insulin resistance. People with FPL are at increased risk of acute pancreatitis in addition to other long-term, progressive manifestations, such as premature cardiomyopathy, atherosclerosis, and liver disease. Additional information on FPL is available through Lipodystrophy United at www.lipodystrophyunited.org.
ABOUT AKCEA THERAPEUTICS
ABOUT IONIS PHARMACEUTICALS
SOURCE: Akcea Therapeutics