The UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) has granted a positive opinion to Akcea Therapeutics’ volanesorsen for the treatment of familial chylomicronaemia syndrome (FCS) under the Early Access to Medicines Scheme (EAMS).
Approval
This decision means that eligible FCS patients will be able to access volanesorsen before the European Commission (EC) makes a formal decision for its use in Europe.
“The MHRA decision is an important development for people with FCS, a condition that is often misunderstood and misdiagnosed,” commented Jill Prawer, chair of the LPLD Alliance. “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.”
FCS is a rare genetic lipid disorder, characterised by extremely high levels of triglycerides. Common symptoms include abdominal pain, and the risk of recurrent, potentially fatal, acute pancreatitis. There is currently no available effective therapy for FCS, the only option for patients is to adopt a severely restricted low fat diet, which does not reduce the levels of triglycerides to ‘normal’ nor does it remove the risk of pancreatitis for most patients.
“We are delighted with the MHRA's decision to give people with FCS early access to volanesorsen,” said Luke Robinson, general manager, Akcea Therapeutics, UK, Ireland & Nordics. “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.”
“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,” added Dr Louis O'Dea, chief medical officer, Akcea Therapeutics. “The MHRA positive opinion is an additional step toward helping people with FCS in their daily struggle to manage their disease.”
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.