MHRA grants UK patients early access to treatment for painful genetic disorder

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Chanice Henry

A treatment for a rare and debilitating genetic lipid disorder has been awarded a place on the Early Access to Medicines Scheme by the MHRA.  

The decision means that eligible patients with familial chylomicronaemia syndrome (FCS )will be able to access volanesorsen, before the European Commission (EC) makes a formal decision for its use in Europe.

Luke Robinson, General Manager of Akcea Therapeutics, UK – the medicine’s manufacturer said: “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,”

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]

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. Even when strictly adhered to, this diet does not decrease triglycerides to normal levels or remove the threat of pancreatitis for most patients.[2 Acute pancreatitis can result in hospitalisation and be life threatening.

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

 “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.

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.

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.

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.

Last month the MHRA granted the 50th Promising Innovative Medicine (PIM) designation as part of the EAMS programme.

In a statement, Dr Ian Hudson, Chief Executive Officer at MHRA said: “Reaching this milestone means 50 new indications from promising medicinal products have emerged in the last four years alone. As a result, current and future patients will be able to access potentially life-saving drugs that they may not have otherwise had the chance to have.”

[1] Brunzell JD. Familial lipoprotein lipase deficiency. GeneReviews 2011.
2] 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