MHRA Grants UK Patients Early Access to Akcea Therapeutics’ Volanesorsen for Familial Chylomicronaemia Syndrome (FCS)
LONDON, March 21, 2018 (GLOBE NEWSWIRE) -- Akcea Therapeutics United Kingdom (UK) Ltd today announced that volanesorsen has been granted a positive scientific opinion through the Early Access to Medicines Scheme (EAMS) by the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA), for the treatment of familial chylomicronaemia syndrome (FCS), a rare genetic lipid disorder. The decision means that eligible patients with FCS will be able to access volanesorsen before the European Commission (EC) makes a formal decision for its use in Europe. Akcea Therapeutics UK, the UK subsidiary of Akcea Therapeutics Inc. (NASDAQ:AKCA), an affiliate of Ionis Pharmaceuticals, Inc., is focused on developing and commercialising drugs to treat patients with serious cardiometabolic lipid disorders.
“The MHRA decision is an important development for people with FCS, a condition that is often misunderstood and misdiagnosed. FCS has an enormous impact on most areas of daily life for people affected by it, including severe restrictions on what they can eat, their relationships, their social life, their employment opportunities and their emotional well-being” comments Jill Prawer, Chair of the LPLD Alliance.
FCS is a severe, rare disorder characterised by extremely high levels of triglycerides, daily symptoms such as abdominal pain, and the risk of recurrent, potentially fatal, acute pancreatitis.1 People with FCS are unable to effectively metabolise large, triglyceride-rich lipid particles called chylomicrons due to a deficiency in lipoprotein lipase, an enzyme that helps to break down triglycerides.1 There is currently no effective therapy available for FCS. The only option people with FCS have is to adopt a severely restricted low fat diet2 of 10-20 grams of fat a day, which, even when strictly adhered to, does not decrease triglycerides to normal levels or remove the threat of pancreatitis for most patients.3
“We are delighted with the MHRA’s decision to give people with FCS early access to volanesorsen. With no current treatment options for people living with this debilitating disease, this will enable them to access and benefit from a new innovative therapy to help address a clear unmet need,” said Luke Robinson, General Manager, Akcea Therapeutics, UK, Ireland & Nordics.
“Akcea is committed to transforming the lives of people with serious cardiometabolic diseases caused by lipid disorders. Even when people with FCS adhere to the restricted diet, this does not necessarily protect them from acute pancreatitis, which can result in hospitalisation and be life threatening. The MHRA positive opinion is an additional step toward helping people with FCS in their daily struggle to manage their disease,” said Dr Louis O’Dea, Chief Medical Officer, Akcea Therapeutics.
EAMS is a UK-based early access programme that provides early availability of innovative and new unlicensed medicines to UK patients with a high unmet clinical need. The medicines approved for the scheme are intended to treat, diagnose or prevent seriously debilitating or life-threatening conditions where no adequate treatment options exist.4 The inclusion of volanesorsen recognises the debilitating nature of FCS and the impact it has on daily living with the absence of adequate treatment options.
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Notes to Editors
About Volanesorsen in FCS
Volanesorsen, a product of Ionis’ proprietary antisense technology, is in development for rare metabolic disorders including familial chylomicronaemia syndrome (FCS). 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 characterised by extremely elevated levels of triglycerides, symptoms such as abdominal pain that affect daily living, and the risk of recurrent, potentially fatal, acute pancreatitis.1 People with FCS are unable to effectively metabolise large, triglyceride-rich lipid particles called chylomicrons due to a deficiency in lipoprotein lipase, an enzyme that helps to break down triglycerides.1 Patients with FCS have triglyceride levels that can reach 20 to 30 times that of healthy individuals,5 which can often lead to repeated episodes of acute pancreatitis, which can be fatal.1 There is currently no effective therapy available. 2
About Akcea Therapeutics UK
Akcea Therapeutics UK is an affiliate of Akcea Therapeutics Inc. Akcea is a biopharmaceutical company focused on transforming the lives of patients with serious cardiometabolic lipid disorders. Akcea has a robust portfolio of development-stage drugs covering multiple targets and diseases using advanced RNA-targeted antisense therapeutics.
Akcea, an affiliate of Ionis Pharmaceuticals, Inc., is advancing a mature pipeline of four novel drugs, including volanesorsen, AKCEA-APO(a)-LRx, AKCEA-ANGPTL3-LRx and AKCEA-APOCIII-LRx, all with the potential to treat multiple diseases. All four drugs were discovered by and are being co-developed with Ionis, a leader in antisense therapeutics, and are based on Ionis’ proprietary antisense technology. The most advanced drug in its pipeline, volanesorsen, is under regulatory review in the U.S., EU and Canada for the treatment of familial chylomicronaemia syndrome, or FCS. Akcea is building the infrastructure to commercialise its drugs globally with a focus on lipid specialists as the primary call point. Akcea’s global headquarters is located in Cambridge, Massachusetts. Additional information about Akcea is available at www.akceatx.com.
Forward Looking Statement
This press release includes forward-looking statements regarding the business of Akcea Therapeutics, Inc., Akcea Therapeutics UK, and the therapeutic and commercial potential of volanesorsen and other products in development. Any statement describing Akcea’s goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavour of building a business around such drugs. Akcea’s forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Akcea’s forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Akcea. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Akcea’s programs are described in additional detail in its annual report on Form 10-K for the year ended December 31, 2017, which is on file with the SEC.
In this press release, unless the context requires otherwise, “Ionis”, “Akcea,” “Company,” “Companies” “we,” “our,” and “us” refers to Ionis Pharmaceuticals and/or Akcea Therapeutics.
Ionis Pharmaceuticals™ is a trademark of Ionis Pharmaceuticals, Inc. Akcea Therapeutics™ is a trademark of Ionis Pharmaceuticals, Inc.
1 Brunzell JD. Familial lipoprotein lipase deficiency. GeneReviews 2011.
2 NORD. The physician’s guide to lipoprotein lipase deficiency (LPLD). Available at: www.rareconnect.org/uploads/documents/nord-physician-guide-to-lipoprotein-lipase-deficiency-lpld.pdf. Last accessed December 2017
3 Davidson M et al. The burden of familial chylomicronaemia syndrome: interim results from the IN-FOCUS study. Expert Review of Cardiovascular Therapy 2017; 15(5):415-423
4 Medicines and Healthcare Products Regulatory Agency Guidance. Apply for the early access to medicines scheme (EAMS). Available at: www.gov.uk/guidance/apply-for-the-early-access-to-medicines-scheme-eams. Last accessed December 2017.
5 Gaudet D, et al. Targeting APOC3 in the familial chylomicronemia syndrome. New England Journal of Medicine 2014; 371:2200-2206