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NICE recommends AMVUTTRA®▼ (vutrisiran) as treatment for a hereditary form of amyloidosis in draft guidance

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MAIDENHEAD, UK, 19th January 2023 – Alnylam UK Limited, the leading RNA interference (RNAi) therapeutics company, today welcomed a draft decision from the National Institute for Health and Care Excellence (NICE) recommending the use of AMVUTTRA® (vutrisiran) on the NHS in England as an option for treating hereditary transthyretin-related (ATTRv) amyloidosis.[ii] Vutrisiran, a subcutaneous injection administered once every three months, seeks to address the root cause of ATTRv amyloidosis by reducing the production of the abnormal protein (amyloid) that drives this disease. Following final guidance, patients will have the option of vutrisiran, meaning that many may now only need to visit the hospital for treatment every three months, compared to existing therapies that need to be administered every few weeks or more frequently. The guidance is expected to be implemented in Wales in due course.


Professor Julian Gillmore, Consultant at the UCL National Amyloidosis Centre, at the Royal Free Hospital, London, said: “Today’s recommendation of vutrisiran marks another remarkable step forward for patients with hereditary ATTR amyloidosis. This medicine targets the production of amyloid at the source – an approach that we already know can be highly effective at slowing down the symptoms of this condition. Now, we have the ability to do this with an injection that can be given every few months  rather than every other week.”


In cases of ATTRv amyloidosis, one of the proteins that is made by the body is not produced correctly. The protein, which is called transthyretin (TTR), can become abnormal in ATTRv amyloidosis, breaking apart into smaller pieces (amyloid). These deposits can build up in different organs and tissues in the body, such as the nerves, heart, eyes, and gastrointestinal tract, causing damage to them and creating potentially life-threatening health problems. When left untreated, patients can develop polyneuropathy (disease of the peripheral nerves), which can result in a loss of sensation in the lower limbs and hands, autonomic dysfunction (affecting the heart, bladder and intestines, among other organs) and loss of mobility.[iii]


Vince Nicholas, Trustee at the UK ATTR Amyloidosis Patients’ Association (UKATPA), said: “Living with ATTR amyloidosis results in life being incredibly difficult. The symptoms of the disease may strip away the independence of the patient and negatively affects their lives and that of their carers. Patients have to attend numerous appointments in a variety of hospitals depending on the medical input they require. Over the last few years, the amyloidosis community has been very fortunate to have seen the introduction of new medicines funded by the NHS that have greatly improved the future for patients with this debilitating disease. This latest approval has the potential to be a turning point for patients, resulting in fewer treatments and much less time in hospital, leaving more of their time to spend doing what matters most to them.”    


Vutrisiran is an RNAi therapeutic that inhibits the production of the TTR protein by the liver, leading to a reduction in the level of TTR in the blood. By preventing the formation of this protein in patients, it is possible to slow or stop the course of disease, avoiding further damage to patients’ organs and bodily tissues.


Philip Davey, Country Manager of Alnylam Pharmaceuticals, UK and Ireland said: “We are delighted that NICE recognised the potential of vutrisiran and that it will now become one of the first rare disease treatments to become available to patients as a result of this accelerated scheme. Throughout the process, we have collaborated closely with NICE to be able to bring this treatment to eligible patients within a much shorter timeframe. Our hope is that it will offer benefits both for patients suffering from this devasting condition, and for healthcare professionals who provide them with vital care. Our first medicine was recommended for reimbursement on the NHS in England for the treatment of ATTRv amyloidosis in 2019, and this was a moment of great pride for us. Securing this latest guidance for this new advanced medicine would take this pride one step further, demonstrating our commitment to deliver innovative medicines for patients who need them.”



[ii] NICE. Vutrisiran for treating hereditary transthyretin-related amyloidosis [ID5074]. Last accessed: January 2023.

[iii] Adams, D., Algalarrondo, V., Polydefkis, M. et al. Expert opinion on monitoring symptomatic hereditary transthyretin-mediated amyloidosis and assessment of disease progression. Orphanet J Rare Dis. 2021;16:411. Last accessed: January 2023.

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