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NICE recommends Alnylam's Onpattro for inherited hATTR amyloidosis

Published on 09/07/19 at 12:03pm

It's good news for UK patients as it emerges that the National Institute of Health and Care Excellence has passed positive recommendation for Alnylam’s Onpattro (patisiran) for the treatment of inherited hATTR amyloidosis, a progressive disease related to the production of the abnormal protein transtyretin which can lead to nerve and organ damage and even death.

The decision means that patients in England and Wales will now be able to access the medicine via the NHS. There are around 150 people affected in the UK.

The recommendation was driven by clinical trial results which demonstrated that Onpattro improved neuropathy symptoms in 52% of 225 hATTR patients with polyneuropathy, compared to just 4% with placebo.

Though the cost of the drug was argued to come in above what the organisation would normally deem cost-effective, the recommendation was ultimately made when taking into consideration “the rarity and severity of the condition, the considerable effect the condition has on families and carers, the size of the health benefits provided by patisiran, and the unique and innovative nature of the treatment.”

“Today's decision is an important step forward in treating a life-threatening and disastrous life-threatening illness. Patisiran in its main clinical study has shown that it can to stop or even improve the potentially debilitating symptoms of this disease in most patients,” commented Professor Philip Hawkins, Head of the National Center for Amyloidosis at the Royal Free Hospital in London. “This means that we now have a real opportunity to maintain the quality of life of eligible patients longer than as far as possible. Gene preservation is a promising area of ​​medicine and is encouraging to see this science that translates into treatments that potentially can help those suffering from serious illnesses such as amyloidosis of HATTR. "

Brendan Martin, UK and Ireland General Manager at Alnylam, also remarked: "Over the past year, Alnylam and NICE have worked together to provide access to the NHS patented in England. We are pleased with today's outcome, which we hope now will help many people affected by this catastrophic disease gain more control over their daily lives. At Alnylam, our goal continues to take precedent science into RNAi and transform this into new drugs that can improve the lives of patients affected by with erosive diseases. Today is another important step in that journey."

Matt Fellows

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