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

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

The resolution signifies that eligible patients with familial chylomicronaemia syndrome (FCS )shall be ready to access volanesorsen, earlier than the European Commission (EC) makes a proper resolution for its use in Europe.

Luke Robinson, General Manager of Akcea Therapeutics, UK – the drugs’s producer mentioned: “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 extreme, uncommon disorder characterised by extraordinarily excessive ranges of triglycerides, daily signs similar to belly ache, and the danger of recurrent, doubtlessly deadly, acute pancreatitis.[1]

There is at present no efficient remedy out there for FCS.

The only option people with FCS have is to adopt a severely restricted low fat diet of 10-20 grams of fats a day. Even when strictly adhered to, this eating regimen doesn’t lower triglycerides to regular ranges or take away the specter of pancreatitis for most patients.[2 Acute pancreatitis may end up in hospitalisation and be life threatening.

People with FCS are unable to successfully metabolise massive, triglyceride-rich lipid particles referred to as 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” feedback Jill Prawer, Chair of the LPLD Alliance.

Akcea Therapeutics UK, the UK subsidiary of Akcea Therapeutics Inc, an affiliate of Ionis Pharmaceuticals, Inc., is targeted on creating and commercialising medicine to deal with patients with severe cardiometabolic lipid problems.

EAMS is a UK-based early access programme that gives early availability of progressive and new unlicensed medicines to UK patients with a excessive unmet scientific want. The medicines permitted for the scheme are supposed to deal with, diagnose or stop critically debilitating or life-threatening circumstances the place no enough treatment choices exist.

The inclusion of volanesorsen recognises the debilitating nature of FCS and the impression it has on daily residing with the absence of enough treatment choices.

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

In an announcement, Dr Ian Hudson, Chief Executive Officer at MHRA mentioned: “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 outcomes from the IN-FOCUS research. Expert Review of Cardiovascular Therapy 2017; 15(5):415-423

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