Akcea Therapeutics has announced that volanesorsen has been granted a positive scientific opinion through the Early Access to Medicines Scheme 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 makes a formal decision for its use in Europe. Akcea Therapeutics UK an affiliate of Ionis Pharmaceuticals is focused on developing and commercialising drugs to treat patients with serious cardiometabolic lipid disorders.
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. 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. There is currently no effective therapy available for FCS. The only option people with FCS have is to adopt a severely restricted low fat diet 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.
“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.
“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.