Akcea Therapeutics has 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, 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 recognition of the innovation that volanesorsen represents for 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.
- Brunzell JD. Familial lipoprotein lipase deficiency. GeneReviews 2011.
- 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
- 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
- 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.