Akcea Therapeutics’ volanesorsen will be available to eligible patients in the UK before it is officially licensed in the EU, having been granted entry to the country’s Early Access to Medicines Scheme.
The Medicines and Healthcare products Regulatory Agency awarded the drug a positive scientific opinion for the treatment of familial chylomicronaemia syndrome (FCS), a rare genetic lipid disorder.
FCS is a severe disorder characterised by extremely high levels of triglycerides, daily symptoms including abdominal pain, and risk of recurrent and potentially fatal acute pancreatitis.
People with the condition are unable to metabolise large triglyceride-rich lipid particles called chylomicrons due to a deficiency in lipoprotein lipase, an enzyme that helps to break down triglycerides.
Currently, the only option for patients is 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, a company spokesperson noted.
“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” said Jill Prawer, chair of the LPLD Alliance.
“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,” added Luke Robinson, general manager, Akcea Therapeutics, UK, Ireland & Nordics.