The UK's Medicines and Healthcare Products Regulatory Agency (MHRA) has granted Akcea Therapeutics' volanesorsen a positive scientific opinion through the Early Access to Medicines Scheme (EAMS) for the treatment of familial chylomicronaemia syndrome (FCS), the company announced on Wednesday.
FCS is a severe and rare disorder that is characterised by extremely high levels of triglycerides. Daily symptoms include abdominal pain, and the risk of recurrent, potentially fatal, acute pancreatitis.
There is currently no effective treatment available for FCS, and the only option for people with FCS is to adopt a severely restricted low fat diet of 10-20 grams of fat a day.
The decision from the MHRA means that eligible patients with FCS will be able to access volanesorsen before the European Commission makes a formal decision regarding its use in Europe.
EAMS is a UK-based early access programme providing early availability of innovative and new, unlicensed medicines to patients in the UK with a high unmet clinical need.
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