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Akcea seeks Health Canada approval for volanesorsen to treat familial chylomicronemia syndrome

Cambridge, Massachusetts
Wednesday, September 13, 2017, 17:00 Hrs  [IST]

Akcea Therapeutics, an affiliate of Ionis Pharmaceuticals, focused on developing and commercializing drugs to treat patients with serious cardiometabolic diseases caused by lipid disorders, and Ionis, announced the filing of a New Drug Submission (NDS) to Health Canada for volanesorsen, an investigational medicine for the treatment of familial chylomicronemia syndrome (FCS). Health Canada has also granted priority review for the volanesorsen NDS. Priority review provides for the "fast-tracking" of eligible regulatory filings in Canada intended for the treatment, prevention or diagnosis of serious, life-threatening or severely debilitating diseases or conditions.

“We are pleased to have completed our third global regulatory filing for volanesorsen. This is a tremendous achievement for Akcea and Ionis, and I would like to express my thanks to all those who helped us get to this place. We are also pleased that Health Canada has granted priority review for the volanesorsen NDS for the treatment of FCS in Canada, which could accelerate access to this important new medicine for Canadians suffering with FCS,” said Paula Soteropolous, president and chief executive officer of Akcea. “With regulatory filings for marketing authorization for volanesorsen submitted in the US, EU and now Canada, we are on track for a potential global commercial launch of volanesorsen in 2018.”

FCS is a severe, rare disorder characterized by extremely high levels of triglycerides, symptoms such as abdominal pain that affect daily living, and the risk of recurrent, potentially fatal, acute 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 have triglyceride levels that can reach 20 to 30 times those of healthy individuals.  This predisposes them to episodes of acute pancreatitis, which is potentially fatal,” said Dr. Robert Hegele, distinguished professor of medicine and biochemistry, Western University and the director of Lipid Genetics Clinic and staff endocrinologist at the London Health Sciences Centre. “Today, there is no effective therapy for FCS patients, so I’m encouraged that we are now very close to having, for the first time, a therapeutic option for FCS patients that can substantially reduce triglycerides to levels that markedly reduce the risk for pancreatitis and could relieve some of the other symptoms that FCS patients live with on a daily basis.”

“We are encouraged that Health Canada has granted priority status to volanesorsen, thereby recognizing the serious, severely debilitating nature of FCS and the potential role this new therapy could play,” says Durhane Wong-Rieger, president of the Canadian Organization of Rare Disorders and chair of the Canadian Heart Patient Alliance. “I have met several individuals with FCS, and have heard many of their stories of intense pain, their utter lack of control in preventing an attack and their fear of the inevitable consequences of their disease.”

Volanesorsen, a product of Ionis’ proprietary antisense technology, is in development for two rare metabolic disorders: FCS and FPL. Volanesorsen is designed to reduce the production of ApoC-III, a protein produced in the liver that plays a central role in the regulation of plasma triglycerides and may also affect other metabolic parameters.

FCS is a severe, rare disorder characterized by extremely high levels of triglycerides, daily symptoms such as abdominal pain, and the risk of recurrent, potentially fatal, acute 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.

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.

 

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